newUnderlying technologies include: Sequencing Technology, Gene Editing (i.e. CRISPR), and Immunotherapy. — Ark Invest, 10h ago
newGenetic Manipulation: With the rapid advancements in CRISPR technology, having access to a vast DNA database might allow for mass genetic modifications. — Conservative Firing Line, 16h ago
newFor malaria infection screening and plasmodium genotyping, the microfluidic platform combined recombinase polymerase amplification (RPA) with clustered regularly interspaced short palindromic repeats (CRISPR)-based detection. — The Guardian Nigeria News - Nigeria and World News, 18h ago
new...“For some hereditary, rare diseases, there is currently no cure. However, gene therapy is a possible solution, and we are now testing various strategies using gene therapy,” Magnar Bjørås says. — sciencenorway.no, 16h ago
David R. Liu’s research integrates chemistry and evolution to illuminate biology and enable next-generation therapeutics. His major research interests include the engineering, evolution, and in vivo delivery of genome editing proteins such as base editors and prime editors to study and treat genetic diseases; the evolution of proteins with novel therapeutic potential using phage-assisted continuous evolution (PACE); and the discovery of bioactive synthetic small molecules and synthetic polymers using DNA-templated organic synthesis and DNA-encoded libraries. Base editing—the first general method to perform precision gene editing without double-stranded breaks, and a Science 2017 Breakthrough of the Year finalist—as well as prime editing, PACE, and DNA-templated synthesis are four examples of technologies pioneered in his laboratory. These technologies are used by thousands of labs around the world and have enabled the study and potential treatment of many genetic diseases. Four base editing clinical trials are already underway to treat leukemia, hypercholesterolemia, sickle-cell disease, and beta-thalassemia, and the first clinical benefit of a base edited therapeutic in a T-cell leukemia trial has been reported. — Events & Lectures, 4d ago
CRISPR, an acronym for clustered regularly interspaced short palindromic repeats, is a family of genes that first evolved in prokaryotic organisms such as bacteria and archaea to defend against infectious phages.1 Analogous to eukaryotic adaptive immune memory, CRISPR sequences derive from bacteriophages that previously infected prokaryotes; bacteria use their CRISPR sequences and nucleases called CRISPR associated (Cas) proteins to detect and destroy familiar bacteriophages.1 Today, researchers build on the mechanisms of prokaryotic CRISPR systems to engineer CRISPR-Cas mediated gene editing technologies, which use Cas proteins and guide RNAs to block, cut, or edit target genes.1... — The Scientist Magazine®, 5d ago
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CRISPR is a gene-editing tool that acts like genetic scissors, paving the way for scientists to edit DNA precisely. The technology involved two essential components – a guide RNA to match a desired target gene and Cas9 (CRISPR-associated protein 9)—an endonuclease that causes a double-stranded DNA break, allowing modifications to the genome according to the National Library of Medicine. — interestingengineering.com, 7d ago
newAlthough RNA-based therapeutics are still a relatively new class of medicines, scientists have been studying the clinical use of ribonucleic acid (RNA) for years. Beyond messenger RNA (mRNA), other categories of RNA-based therapeutics have long been established and approved for use, including antisense oligonucleotides (ASO), RNA interference (RNAi) and RNA aptamers. However, the success of using mRNA-based vaccines for Covid-19 spurred public awareness about mRNA’s disruptive potential in medicine. — MedCity News, 2d ago
Experts at Penn Medicine are researching novel treatments for heart disease, including CRISPR gene editing technology, CAR T technology, and mRNA injections. — Penn Today, 4d ago
...“CRISPR Therapeutics is a clinical-stage gene editing company focused on the development of CRISPR/Cas9-based therapeutics. The company’s proprietary platform specializes in Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9, which precisely cuts DNA to disrupt, delete, correct, and insert genes to treat genetically defined diseases. — Morningstar, Inc., 11w ago
Gene Therapy and Gene Editing: Gene therapy and gene editing technologies hold great promise for the treatment of genetic disorders. Gene therapy involves introducing therapeutic genes into a patient's cells to correct genetic abnormalities, while gene editing techniques like CRISPR-Cas9 enable precise modification of DNA sequences to fix or remove disease-causing mutations. These approaches have the potential to provide long-term, curative treatments for previously untreatable genetic diseases. — marketsandmarkets.com, 6w ago
Recent breakthroughs in RNA therapeutics from CRISPR-based RNA editing to antisense oligonucleotides hold promise for patients. These technologies allow researchers to mute RNA transcripts with gene mutations or correct mutations directly. — STAT, 4w ago
In 2012, George Church, Jennifer Doudna, Emmanuelle Charpentier, and Feng Zhang discovered that by designing guide RNA to target a specific region in the genome, “the CRISPR-Cas9 system can be used as a “cut-and-paste” tool to modify genomes. As a DNA-editing tool, CRISPR-Cas9 can remove or introduce new genes as well as silence or activate genes. CRISPR-Cas9 has been used to switch off genes that limit the production of lipids in microalgae, leading to increased lipid production and higher yields of biofuel. [9] In the near future, CRISPR-Cas9 may also be used to cure genetic disorders such as sickle-cell anemia and cystic fibrosis. [10] In fact, there is already a wide range of CRISPR applications in disease treatment, including cancer and infectious diseases. — Bitesize Bio, 24d ago
Gene Editing and Gene Therapies: Advancements in gene editing technologies like CRISPR-Cas9 have opened up new possibilities for targeted interventions at the genetic level. Gene therapies hold potential for treating genetic disorders, rare diseases, and certain types of cancers by correcting or modifying defective genes. — marketsandmarkets.com, 6w ago
One of the most transformative biotech breakthroughs in recent years has been the development of CRISPR–-Cas9 gene editing technology. CRISPR, short for clustered regularly interspaced short palindromic repeats, has revolutionized genetic engineering by introducing the ability to alter an organism’s DNA to precision medicine. This revolutionary technique allows scientists to precisely edit genes, opening new possibilities for treating genetic diseases and advancing biotechnology. — LifeSciencesIntelligence, 6w ago
Optical tweezers are endowed with DNA identification through CRISPR-based biosensing systems as an avenue for biomolecule enrichment to cleave the CRISPR complex. Such CRISPR-powered optothermal nanotweezers or CRONT systems hold immense promise to advance the understanding of complex biological processes as a versatile detection probe across biomedical research, drug discovery, and disease diagnostics. — phys.org, 6d ago
Best known as a powerful gene-editing tool, CRISPR actually comes from an inbuilt defense system found in bacteria and simple microbes called archaea. CRISPR systems include pairs of "molecular scissors" called Cas enzymes, which allow microbes to cut up the DNA of viruses that attack them. CRISPR technology takes advantage of these scissors to cut genes out of DNA and paste new genes in. — livescience.com, 5d ago
Synthego is a genome engineering company focused on making gene editing technologies more accessible to researchers and scientists. Their approach combines machine learning, automation, and gene editing, aiming to speed up life science research and development. Synthego’s platforms offer large-scale and high-quality gene editing tools and cell-based models, which are essential for scientific discoveries and clinical trials. The company’s mission is to simplify genome engineering, allowing scientific researchers to focus more on discovery and development, and less on the complexities of the tools required. — InsideScientific, 5d ago
CRISPR systems have been leveraged to develop biomolecular approaches, such as CRISPR-Cas-mediated genome editing. The discovery of previously unknown CRISPR systems has potential to lead to the further development of these biotechnologies, like safer and more effective genomic therapeutics. — Drug Target Review, 10d ago
newThe team then applied novel genomic tools to investigate how the otter population changed in Britain, using whole genome DNA sequencing. — phys.org, 2d ago
A few adeno-associated virus-delivered gene therapies have been approved by the Food and Drug Administration to treat genetic diseases such as spinal muscular atrophy and Hemophilia B. However, few researchers have explored using this approach against tumors. The authors believe this study and other work demonstrate the potential of gene therapy for cancer treatments. — medicalxpress.com, 3d ago
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Gene Editing and Gene Therapy: Research is ongoing in the field of gene editing and gene therapy to address the root cause of CF by repairing or replacing the defective CFTR gene. Techniques such as CRISPR/Cas9 and viral vectors for gene delivery hold promise in this area. — WriteUpCafe.com, 14d ago
Wave Life Sciences Ltd. (NASDAQ: WVE) is a clinical-stage genetic medicine company that specializes in the design, optimization, and creation of cutting-edge stereopure oligonucleotides (short, single- or double-stranded DNA or RNA molecules designed to pair with a strand of DNA or RNA to modulate gene and protein expression), aiming to combat diseases with few treatment alternatives, with lead programs addressing Duchenne muscular dystrophy, Huntington’s disease, and alpha-1 antitrypsin deficiency. — Finbold, 11w ago
CRISPR and CRISPR-Associated (Cas) Genes is a genome editing tool that enables the researchers to make changes in the DNA. CRISPR-Cas9 stands for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9. In recent years the CRISPR and CRISPR-Associated (Cas) Genes has gained lot of popularity as it offers it is cheaper, faster, accurate, and more efficient genome editing methods. — openPR.com, 25d ago
The therapy, named EBT-101, involves using CRISPR-Cas9 gene editing to treat HIV. This potential treatment strategy has been studied in animal models since the development of CRISPR-Cas9 in 2012. However, this is the first time such a gene-editing treatment for HIV has been tried in humans. The latest data from the trial suggest that EBT-101 is safe at the doses tested, but we don't yet know if it cures HIV. — livescience.com, 3d ago
The growing space of therapeutic gene editing technologies including CRISPR-Cas9 is helping to facilitate the investigation into molecular pathways involved in disease. In this session, Neil discussed the use of CRISPR-Cas9 in drug development, as well and the challenges associated with method development and their potential bioanalytical solutions. The main message of the session focused around the shift of CRISPR-Cas9 from ‘bench to bedside’ and offering disease treatment via permanent gene editing. — Bioanalysis Zone, 4d ago
New programmable gene editing proteins found outside of CRISPR systems... — phys.org, 9d ago
Biotechnology stocks frequently draw substantial investor interest, and Editas Medicine, Inc. (NASDAQ: EDIT) is no exception. It is a clinical-stage biotechnology company developing therapies for rare diseases based on CRISPR gene editing technology. — Finbold, 4d ago
An early-stage clinical trial raises hope for a new, single-dose HIV therapy that uses CRISPR, the famous gene-editing system. — livescience.com, 4d ago
In this study, the researchers aimed to investigate the relationship between gene transcription and chromatin accessibility, which denotes the extent to which chromatin is open and accessible for gene regulation and subsequent transcription. They used advanced techniques such as ATAC-seq (assay for transposase-accessible chromatin sequencing) and snRNA-seq (single-nucleus RNA sequencing). — What is Epigenetics?, 5d ago
Scientists have touted RNA editing as a safer derivation of early gene editing methods. Unlike CRISPR-based therapies, RNA editing can target specific sites in an RNA transcript without permanently changing a patient's genome. — BioPharma Dive, 10w ago
...“Viral vector deliveries of genetic tools of any kind is the future of medicine,” says Paul Garofolo, cofounder and CEO of Locus Biosciences, a clinical-stage biotech company developing phage therapies based on CRISPR-Cas3, which chews back long stretches of DNA. Locus Biosciences is developing multiple phages as precision therapeutics to target E. coli urinary tract infections and infections with P. aeruginosa, S. aureus, and K. pneumoniae. Their CRISPR-enhanced bacteriophage cocktail (crPhage®) to treat urinary tract infections caused by E. coli is the first randomized Phase 2/3 clinical trial of CRISPR-enhanced bacteriophage to treat bacterial infections. — synbiobeta.com, 7w ago
...like CRISPR-Cas9 hold the potential to correct genetic defects, offering... — omicsonline.org, 9w ago
Advancements in CRISPR Technology: CRISPR-Cas9 technology has revolutionized gene editing, enabling precise and efficient modifications in the genome. — WriteUpCafe.com, 10w ago
A virus-free, CRISPR-based gene-editing technique that has shown promise in rodent models is being developed for use in people with Angelman syndrome or HIST1H1E syndrome. The technique — Stimuli-responsive Traceless Engineering Platform, or STEP — uses engineered bundles of protein and RNA called ribonucleoproteins to penetrate neurons and deliver gene therapy throughout the brain. — Spectrum | Autism Research News, 5w ago
PORTLAND, OREGON, UNITED STATES, October 17, 2023 /EINPresswire.com/ -- Gene editing market was valued at $3.9 billion in 2021, and is estimated to reach $7.4 billion by 2031, growing at a CAGR of 6.7% from 2022 to 2031. Gene editing also known as genome editing, is a field of study that aims to modify genes in live animals in order to better understand gene function and create treatments for hereditary and acquired disorders. In many different types of cells and species, genome editing can be used to fix, introduce, or delete practically any DNA sequence. While DNA editing techniques have been around for decades, new ways have made it faster, cheaper, and more efficient. The revelation that a broken portion of DNA in a gene stimulates a cell's repair system to patch the split together led to the development of genome editing. Researchers can use genome editing to replicate the natural process of DNA repair. Zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and meganucleases are advanced genome editing technologies based on proteins. Another approach is CRISPR/Cas9, which stands for clustered regularly interspaced short palindromic repeats. — EIN Presswire, 6w ago
The research, published in Nature Communications, unravels new potential for simple and sensitive nucleic acid detection in various diagnostic settings—including early cancer diagnostics and infectious disease detection— using new and improved clustered regularly interspaced short palindromic repeats (CRISPR) technology. CRISPR technology uses an enzyme that can be programed to seek out a specific stretch of nucleic acid and grab onto it. — Technology Networks, 6d ago
RNA undergoes extensive modification through enzymatic post-transcriptional editing events. Adenosine-to-inosine (A-to-I) editing is one of the most widespread and impactful of these modifications and is catalyzed by adenosine deaminases acting on RNA (ADARs). Resulting inosines base pair with cytosine, essentially re-coding adenosine sites to guanine. Editing is essential for a number of processes including embryogenesis, neurological function, and innate cellular immunity. Dysfunctional editing is also linked to auto-immune diseases, neurological disorders, and several types of cancer. Despite this importance, numerous challenges remain for studying A-to-I editing, and our overall understanding of the locations and frequency of inosine sites remains limited. To address this challenge, we have repurposed EndoV from an RNA-cleaving enzyme into an RNA-binding protein and demonstrated its use for mapping of A-to-I editing sites and global profiling of RNA inosine content in cells and tissue samples. — rit.edu, 5d ago
The team used an approach called RNA interference (RNAi) to assess the molecular pathways influencing the survival of senescent cells. RNAi prevents protein production by reducing gene expression. — medicalxpress.com, 4d ago
The CRISPR-Cas9 gene-editing tool is one of the most important scientific developments of the past decade, earning its discoverers a Nobel Prize in Chemistry. Scientists can use it to make efficient cut-and-paste edits to human cells, potentially treating a huge range of diseases, as well as improving crops, controlling pests, and manipulating bacteria. — New Atlas, 7d ago
In addition to genetics research and genetically engineering in agriculture, CRISPR hold the promise of treating, and potentially even curing, genetic diseases. When applied to living organisms, however, there is another challenge. While CRISPR itself can target the desired DNA sequence, you also have to get the CRISPR to the target cells. This is the more challenging aspect of gene therapy – which vectors do we use to deliver the CRISPR? But there are some conditions where we can bring the cells to the CRISPR rather than the CRISPR to the cells – diseases of the blood. This is the case for sickle cell and TDT, which is why these are the diseases targeted by the first approved CRISPR therapy. — Science-Based Medicine, 12d ago
The two partners CRISPR Therapeutics and Vertex have secured the world’s first approval for a genome editing therapy based on what are known as gene scissors for the UK market. CASGEVY can replace stem cell transplants in the case of two hereditary diseases. — S-GE, 13d ago
These findings provide a foundation for correcting genetic mutations in the brain through genome editing in vivo and offer potential treatment options for people with genetic neurodevelopmental disorders. Since base editors only edit regions from C to T and A to G, using base-editing systems that target a wider range of regions, such as prime editing, would greatly assist in creating genetic tools to treat genetic disorders. — GEN - Genetic Engineering and Biotechnology News, 7d ago
The CRISPR system cuts genes out of DNA using an enzyme called Cas9. These "molecular scissors" are guided to target DNA by a molecule of RNA. The technology was adapted from a natural defense mechanism that bacteria and other simple organisms called archaea use against viruses. — livescience.com, 12d ago
Another driver for the market is the advancements in gene editing technologies. Recent developments in gene editing tools such as CRISPR-Cas9 have revolutionized the field of synthetic biology. These tools allow scientists to precisely edit DNA sequences, enabling the creation of new and improved biological systems with increased efficiency and precision. — Medgadget, 14d ago
CRISPR-Based Epigenome Editing Platform Identifies Genes That Improve T-Cell Therapies for Cancer Treatmentbbbb... — Inside Precision Medicine, 18d ago
The research, published in Nature Communications, unravels new potential for simple and sensitive nucleic acid detection in various diagnostic settings—including early cancer diagnostics and infectious disease detection—using new and improved clustered regularly interspaced short palindromic repeats (CRISPR) technology. CRISPR technology uses an enzyme that can be programmed to seek out a specific stretch of nucleic acid and grab onto it. — phys.org, 12d ago
In a paper, "Base editing of the HBG promoter induces potent fetal hemoglobin expression with no detectable off-target mutations in human HSCs," published in Cell Stem Cell, the team compares gene editing techniques while formulating a method that could have important clinical applications. — medicalxpress.com, 9d ago
The facility will provide researchers from across the UK with access to large-scale biological and technological tools and house an advanced automated arrayed-CRISPR screening platform. It is hoped that through the use of tools, such as CRISPR gene editing to provide insights into the relationship between genes and disease, scientists will discover new opportunities to develop therapies for chronic diseases including cardiovascular, respiratory and metabolic disease. — cambridgenetwork.co.uk, 6d ago
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Engineers Devise a Way to Selectively Turn on RNA Therapies in Human Cells... — ScienceDaily, 7d ago
In a similar study, one led by the Innovative Genomics Institute’s Jennifer A. Doudna, PhD, CRISPR-Cas13a was used to introduce markerless genome edits to three diverse phages with 100% efficiency, including multigene deletions and a codon replacement.12 These techniques demonstrate the possibility of using CRISPR-Cas tools to create small, precise genome edits and engineer a phage genome to meet therapeutic specifications. — GEN - Genetic Engineering and Biotechnology News, 19d ago
In parallel, CRISPR gene editing has emerged as an extremely precise method for targeting DNA sequences by exploiting sequence-specific guide RNAs. Although powerful for biosensing, CRISPR techniques have traditionally depended upon slow, random diffusion to bring sparse target DNA strands in contact with CRISPR enzyme complexes in solution. This bottleneck has limited detection sensitivity and hindered CRISPR's use for rapidly screening low abundance mutants. — nanowerk.com, 7d ago
Furthermore, advancements in gene editing technologies, such as CRISPR-Cas9, have provided researchers with precise tools to manipulate epigenetic modifications. These technologies offer the potential to correct aberrant epigenetic marks and restore normal gene expression patterns, paving the way for the development of novel therapies. — Medgadget, 6w ago
As gene editing research advanced, scientists unearthed different techniques for editing DNA as well as other CRISPR-associated enzymes to do the job. — BioPharma Dive, 7w ago
Regeneron Pharmaceuticals, Inc. and Intellia Therapeutics, Inc. expanded their research collaboration to develop additional in vivo CRISPR-based gene editing therapies focused on neurological and muscular diseases. This builds on the success of the companies’ existing collaboration and combines both companies’ biology and technology expertise. The collaboration will leverage Regeneron’s antibody-targeted adeno-associated virus (AAV) vectors and delivery systems and Intellia’s Nme2 CRISPR/Cas9 (Nme2Cas9) systems adapted for viral vector delivery and designed to precisely modify a target gene. — Contract Pharma, 8w ago
A new RNA editing tool is opening up new ways to develop novel RNA therapies, speed up engineering of human viruses and test drugs. In a proof of concept experiment, researchers used a cutting enzyme – an endonuclease – and a programmable RNA repair enzyme to modify an RNA virus genome. — Chemistry World, 9w ago
CRISPR–Cas9 Genome editing in Nothobranchius furzeri for Gene Knockout and Knock-In... — eLife, 5w ago
The gene-editing tool CRISPR has given researchers the ability to replace and alter segments of DNA, and scientists are applying the technology in fields as diverse as malaria prevention and crop production. In more recent years human trials have started, that aim to use CRISPR-based technology to address genetic disorders. — GEN - Genetic Engineering and Biotechnology News, 9w ago
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GEN - Genetic Engineering and Biotechnology News, 7d ago
More of those programs are built around emerging technologies like antisense oligonucleotides, radioligands and cellular therapies. Pluvicto, for instance, is a radioligand therapy for cancer, while Novartis’ new cholesterol drug Leqvio is an antisense therapy. — BioPharma Dive, 6d ago
The field needs better genome sequencing. Once precise whole genome sequencing of sugarcane is successfully obtained, the next step will be to develop techniques capable of making specific modifications in the genome. The main hope for most scientists resides in CRISPR-Cas9 gene editing. — Science 2.0, 7d ago
The FDA certainly recommends biodistribution studies for proposed drugs that involve gene therapy; drugs which include genetic materials such as RNA and messenger RNA (mRNA) (FDA 2023). As a result, Moderna officials would have fully expected that the FDA would regulate their mRNA drugs as gene therapy drugs (SEC 2019). This is why Moderna researchers had performed a biodistribution study in 2017 on their experimental mRNA drug against cytomegalovirus (CMV) (Moderna Therapeutics n.d.). — sgtreport.com, 9d ago
They found several new variants of known Type I CRISPR systems, which use a guide RNA that is 32 base pairs long rather than the 20-nucleotide guide of Cas9. Because of their longer guide RNAs, these Type I systems could potentially be used to develop a more precise gene-editing technology that is less prone to off-target editing. Zhang’s team showed that two of these systems could make short edits in the DNA of human cells. And because these Type I systems are similar in size to CRISPR-Cas9, they could likely be delivered to cells in animals or humans using the same gene-delivery technologies being used today for CRISPR. — GEN - Genetic Engineering and Biotechnology News, 7d ago
These small molecule approaches are not the only potential ways to edit the epigenome. Subhash Pandey, a neuroscientist at the University of Illinois at Chicago, uses CRISPR/dCas9 constructs to target specific histone modifications of genomic regions. — Drug Discovery News, 13d ago
The gene-editing technology CRISPR shows early promise as a therapeutic strategy for the aggressive and difficult-to-treat brain cancer known as primary glioblastoma, according to new findings. — ScienceDaily, 6d ago
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In addition to sickle cell disease, scientists are applying CRISPR-based editing to a range of conditions. This includes Huntington’s disease (HD), an inherited condition that affects nerve cells in the brain. At the University of California, Berkeley (UCB), scientists have used gene editing technology and other tools to edit the mutant HTT gene responsible for the development of the fatal neurodegenerative disease. Details from the study were presented by Camelle Catamura, a computational biology graduate student at UCB. The team’s approach involves deleting sections of HTT gene and replacing them with an engineered DNA sequence to stop the production of abnormal proteins. To be clear, there is still work to be done but strategically editing a target section of a gene to create a new exon is proving to be a way of feasibly limiting or reducing abnormal mRNA expression. — GEN - Genetic Engineering and Biotechnology News, 26d ago
Excision BioTherapeutics is using the gene-editing technology CRISPR to cure HIV. — Big Think, 4w ago
Clustered regularly interspaced short palindromic repeats (CRISPR) is a bacterial defence system that can cut DNA, first discovered in the DNA sequences of Escherichia coli bacteria (Duckett and Koop, 1977). To bind and cut DNA, CRISPR works with a nuclease called Cas and a guide RNA (gRNA) sequence that directs Cas to its target. Since the discovery of its ability to cut DNA, this mechanism has been used in laboratories for basic molecular research and has rapidly developed into a tool for editing genomes (Ran et al., 2013). Nobel laureates Emmanuelle Charpentier and Jennifer Doudna first adapted CRISPR-Cas9 as a gene-editing tool over 10 years ago (Jinek et al., 2012), and the technology has been rapidly developed and used for many different purposes in a wide range of organisms, including humans. We can name RNA editing, base and prime editing, live imaging and diagnostics, and some treatment clinical trials are already underway (Huang et al., 2022; Pisciotta and Pareyson, 2023; Zha et al., 2023). — The Company of Biologists, 6w ago
Advancements in Viral-Vector Therapies: Viral-vector gene therapies, which use modified viruses to deliver therapeutic genes into cells, are a significant area of focus in the gene therapy field. Keeping pace with the increasing demand for viral-vector therapies requires addressing challenges, exploring standardization opportunities, and strategizing to accelerate patient access. — Medgadget, 14d ago
Best Established Biotech Company, kindly sponsored by Precision BioSearch Winner – Touchlight Genetics - A biotechnology CDMO enabling the development of genetic medicines with its enzymatic doggybone DNA technology. — cambridgenetwork.co.uk, 11d ago
RNA Control Switch: Engineers Devise a Way To Selectively Turn On Gene Therapies in Human Cells... — SciTechDaily, 10d ago
CRISPR technology — whose developers won the 2020 Nobel Prize in chemistry — is a gene-editing tool adapted from natural defense mechanisms in bacteria. — Sickle Cell Disease News, 13d ago
Advancing viticulture: Pioneering transgene-free CRISPR genome editing in grapevines... — phys.org, 12d ago
VERVE-101 uses a modified version of CRISPR gene editing, which allows researchers to alter DNA sequences and thus modify gene function. CRISPR gene-editing techniques typically snip through both strands in DNA's twisted double helix and then rely on a cell's repair mechanisms to fix that cut. However, this raises the risk that unwanted mutations will enter the DNA. — livescience.com, 18d ago
July 16, 2020 Cas proteins like CRISPR-Cas9 have great potential for gene therapy to treat human disease and for altering crop genes, but the gene-targeting and gene-cutting Cas proteins are often large and hard ... — ScienceDaily, 6w ago
Several tools exist for researchers to consider in their experimental designs, including RNA interference (RNAi) using small interfering RNAs (siRNA) derived from eukaryotic genomes, transfection of cells with CRISPR-Cas9 cassettes integrated into plasmid or viral vectors and, more recently, the development of chemically synthesized CRISPR guide RNAs (gRNAs) or CRISPR RNPs directly delivered into cells. Each method has its advantages and drawbacks to consider. — Technology Networks, 12w ago
Gene editing is possible with the use of CRISPR technology. CRISPR is a straightforward method for locating a particular DNA sequence inside a cell. Usually, the following step in CRISPR gene editing is to modify that specific DNA sequence. — Science Times, 4w ago
In a groundbreaking development published in Cell, scientists have unveiled a new, compact CRISPR-based gene-editing tool, AsCas12f. Engineered to be one-third the size of the widely-used Cas9, AsCas12f can be packaged more efficiently into adeno-associated viruses (AAVs), which serve as non-harmful carriers for introducing CRISPR enzymes into various cell types. This breakthrough enzyme has the potential to significantly enhance the efficiency and precision of gene editing, offering hope for more effective treatments for patients with genetic disorders. — synbiobeta.com, 9w ago
CRISPR is adept at modifying genetic code to enhance an organism's performance or correct mutations. CRISPR Cas9 requires a guide RNA (gRNA) to direct the enzyme to its target site to perform these modifications. However, existing computational models for predicting effective guide RNAs in CRISPR tools have shown limited efficiency when applied to microbes. ORNL's Synthetic Biology group, led by Carrie Eckert, observed these disparities and set out to bridge the gap. — synbiobeta.com, 23d ago
Fanzors are RNA-guided enzymes that, like CRISPR enzymes, can be programmed to cut DNA at specific sites. Their discovery earlier this year made them the first such enzymes to be found in eukaryotes and sparked hope that a novel technology for human genome editing could be on the horizon. — IFLScience, 7w ago
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Last year, Graphite dosed its first patient with nulabeglogene autogedtemcel (nula-cel, formerly GPH-101), a gene-editing autologous hematopoietic stem cell (HSC) therapy designed to directly correct the genetic mutation that causes sickle cell disease (SCD). Nula-cel used Graphite’s UltraHDR™ gene editing platform, designed to take CRISPR beyond cutting by harnessing the power of high-efficiency precision DNA repair. — GEN - Genetic Engineering and Biotechnology News, 18d ago
CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, is a bioengineering tool that modifies genetic code to improve an organism’s performance and correct mutations. Also, the CRISPR Cas9 tool uses a unique guide RNA that aids the Cas9 enzyme to bind with a genome. — INQUIRER.net, 18d ago
ORNL scientists developed a method that improves the accuracy of the CRISPR Cas9 gene editing tool used to modify microbes for renewable fuels and chemicals production. This research draws on the lab’s expertise in quantum biology, artificial intelligence and synthetic biology... — analytica-world.com, 13d ago
...4. gene editing, where the desired genes are activated using a variety of methods including CRISPR, using only genes within the genome. — interest.co.nz, 11d ago
A new study by Claudia Kutter's research group at the Department of Microbiology, Tumor and Cell Biology (MTC) has identified potential pitfalls in the use of the gene editing technique CRISPR-Cas9, a gene scissors that is used for cancer treatments. — medicalxpress.com, 12d ago
Mana.bio leverages Artificial Intelligence (AI)/ Machine Learning (ML) to design novel non-viral ex-liver cell-specific RNA delivery solutions. Mana Bio is a biotech start-up leveraging data, machine learning, and high throughput screening to design novel LNPs for extrahepatic delivery of nucleic acid therapeutics and vaccines. Mana was founded by a team of serial entrepreneurs and highly regarded experts in the field of drug delivery, machine learning, and software development. — Falling Walls, 11d ago
To minimize off-target effects, researchers from Wuhan University tested a new gene editing approach with a “transformer” base editor (tBE) to optimize the same therapeutic strategy used by Casgevy to treat sickle cell disease and other β-hemoglobinopathies like β-thalassemia. The tBE has a “fuse-like” mechanism that causes the editor to shut off upon executing an edit, allowing on-target editing while significantly reducing off-target editing. Utilization of tBE for the gene editing approach used by Casgevy resulted in undetectable off-target effects and durable hematopoietic stem cells (HSCs) that carried the edit, demonstrating a safe and effective strategy for treating β-hemoglobinopathies. — GEN - Genetic Engineering and Biotechnology News, 13d ago
The imminent approval of the world's first CRISPR treatment for sickle cell disease is just the start: soon this gene-editing tool could be used to tackle everything from cancer to high cholesterol and infertility... — New Scientist, 20d ago
In other news, the RNA editing company Korro Bio and regenerative medicine company Frequency Therapeutics, announced that they have entered into a definitive merger agreement to combine the companies in an all-stock transaction. Korro’s RNA editing platform—Oligonucleotide Promoted Editing of RNA (OPERA)—stems from research in the laboratory of Josh Rosenthal, PhD, a neurobiologist at the Marine Biological Laboratory in Woods Hole, MA. His research into marine organisms, and their adaptation to physical environments, led him to focus on RNA editing. — GEN - Genetic Engineering and Biotechnology News, 20d ago
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GEN - Genetic Engineering and Biotechnology News, 7w ago
Gene therapy has great promise for treating genetic diseases, and even for more common diseases such as atherosclerosis (hardening of the arteries). Over the past decade, the gene-editing technology CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)—a family of DNA sequences found in the genomes of certain bacteria—has allowed scientists to fix individual errors in the genetic code that cause disease. — medicalxpress.com, 17d ago
Genetic engineers commonly use the latest gene editing technologies such as Crispr to selectively modify the genetics of an organism. However, such rational methods require knowledge of what you are targeting and why. Conversely, random mutagenesis can be a useful scatter-gun approach – simply induce mutations in an organism and see what happens. — Chemistry World, 12d ago
Oct. 29, 2020 Researchers have developed CRISPR-LICHT, a revolutionary technology that allows genetic screens in human tissues such as brain organoids. By applying the novel technology to brain organoids, the ... — ScienceDaily, 17d ago
A diverse set of species, from snails to algae to amoebas, make programmable DNA-cutting enzymes called Fanzors — and a new study from scientists at MIT’s McGovern Institute for Brain Research has identified thousands of them. Fanzors are RNA-guided enzymes that can be programmed to cut DNA at specific sites, much like the bacterial enzymes that power the widely used gene-editing system known as CRISPR. The newly recognized diversity of natural Fanzor enzymes, reported recently in the journal Science Advances, gives scientists an extensive set of programmable enzymes that might be adapted into new tools for research or medicine. — SciTechDaily, 23d ago
Researchers devise genetically encoded DNA origami for targeted and precise gene therapy in vivo... — phys.org, 9d ago
Casgevy is a gene-editing tool devised using CRISPR-Cas9 to treat two severe blood conditions – sickle-cell disease and β-thalassaemia. — interestingengineering.com, 17d ago
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In addition, by combining different RNAP variants into the system, they also demonstrated the creation of multilayer biological circuits for either signal amplification or orthogonal signal transduction. Aside from using this TdRNAP system simply for target molecule detection, the research team also demonstrated its application for cell-specific genome editing by using the system to induce guide RNA (gRNA) expression to trigger the CRISPR/Cas9-mediated deletion of a transgene previously incorporated into the genome of an experimental cell line. — phys.org, 13d ago
Casgevy corrects the mutated hemoglobin gene using CRISPR, an easy-to-use gene editing tool that was first discovered in bacteria. The tool works by using a specialized protein, called Cas9, to cut DNA at a specific location, allowing clinicians to remove, add, or alter specific gene sequences. — Inverse, 17d ago
Using a new algorithm called FLSHclust (“flash clust”), researchers have discovered 188 rare and previously unknown CRISPR-linked gene modules – including a novel type VII CRISPR-Cas system – among billions of protein sequences. The approach and its findings provide novel opportunities for harnessing CRISPR systems and understanding the vast functional diversity of microbial proteins. CRISPR systems have been leveraged to develop a growing suite of novel biomolecular approaches, including CRISPR/Cas-mediated genome editing. The discovery of previously unknown CRISPR systems has the potential to lead to the further development of these biotechnologies, including safer and more effective genomic therapeutics. The CRISPR toolbox has been expanded through computational searches of protein sequence databases. However, the algorithmic approaches commonly used have become impractical for mining exponentially growing datasets containing billions of proteins. To address this limitation, Han Altae-Tran and colleagues developed FLSHclust (fast locality-sensitive hashing-based clustering) – an algorithm for clustering proteins by sequence similarity, which, unlike currently available methods, can quickly and efficiently analyze vast protein sequence databases. To evaluate their approach, Altae-Tran et al. used FLSHclust to search for rare CRISPR systems in an 8.8 terrabase pair metagenomic database containing 8 billion proteins and 10.2 million CRISPR arrays. The analysis uncovered 188 previously unknown CRISPR-associated genes. The authors also identified and characterized a new class of Cas-14 containing CRISPR system, type VII, which acts on RNA. According to the findings, the newly identified systems were rare, and many only encompassed a single cluster out of the nearly 130,000 CRISPR-linked clusters revealed by FLSHclust. “The discovery of previously unknown cas genes and CRISPR systems substantially expands the known CRISPR diversity, emphasizing the functional versatility of CRISPR whereby previously undiscovered proteins and domains are often recruited, either replacing preexisting components or conferring newly identified functions to the preexisting scaffold of Cas proteins,” write Altae-Tran et al. “Taken together, the results of the work reveal unprecedented organizational and functional flexibility and modularity of CRISPR systems but also demonstrates that most variants are rare and only found in relatively unusual bacteria and archaea.”... — SCIENMAG: Latest Science and Health News, 10d ago
Otsuka Pharmaceutical Co., Ltd. and Shape, a programmable medicine company using AI and RNA to target genetic diseases, partnered to develop intravitreally delivered adeno-associated viruses (AAVs) for ocular diseases, with options to add additional targets and tissue types. The companies will apply Shape’s AAVid capsid discovery platform and transgene engineering technology with Otsuka’s expertise in genetic payload design and ophthalmology to develop novel treatments for serious eye diseases. — Contract Pharma, 12w ago
Targeting the genetic instructions for disease proteins (ribonucleic acid or “RNA”) using using splicing, editing, and silencing techniques are also a new way GSK is using tech to discover and develop potential new medicines. — gsk.com, 11w ago
Unlike gene editing therapies that modify the DNA of cells in a laboratory, in vivo treatments do their editing work inside the body and are therefore considered more risky. Along with Excision’s therapy, the FDA has given a green light to testing of in vivo treatments from Sangamo Biosciences, Intellia Therapeutics and Verve Therapeutics. (Sangamo has since terminated its study.)... — BioPharma Dive, 5w ago
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing. — gsk.com, 4w ago
A CRISPR-like gene-editing system found in animals and other complex organisms has been used to edit human cells for the first time. — Freethink, 9w ago
The ability to make synthetic DNA and RNA by chemical or enzymatic means has revolutionized genetic research and drug discovery. Antisense technology employs short, chemically synthesized oligonucleotides (antisense oligonucleotides, or ASOs) that are made in an “antisense orientation” to the target cellular RNA so that they will bind or anneal to that RNA and be used to alter gene expression. The method was first described in the late 1970s by Lasker Award winner Paul Zamecnik, PhD, and Mary Stephenson, PhD, who used unmodified DNA ASOs to inhibit viral RNA translation in the Rous sarcoma virus. — GEN - Genetic Engineering and Biotechnology News, 12w ago
CRISPR is a way to change specific areas of DNA. The system was discovered in bacteria, which use it as an immune system to fend off attacks by viruses. Bacteria store parts of their enemy viruses’ DNA so they can cut the viruses’ genes and defend against them. A CRISPR editing system has two parts: a “guide RNA” sequence and a pair of molecular “scissors.” The guide sequence, like a Dewey decimal number for DNA, leads the scissors to a specific spot on the double helix and the scissors cut the strand of DNA there, inactivating that gene. More recent technologies allow CRISPR systems to insert new DNA into this site, or swap out individual nucleotides — the “letters” in the DNA sequence. — STAT, 17d ago
Meanwhile, a researcher in the Netherlands recently announced progress using CRISPR to develop a potato resistant to the devastating potato blight. “With the gene editing technology CRISPR/Cas, he made potato plants resistant to late blight disease caused by Phytophthora infestans. He did so without the insertion of foreign DNA in the potato genome,” reports Wageningen University & Research. — Bio.News, 11d ago
The new treatment uses the CRISPR-Cas9 gene editing technique, which enables scientists to make precise alterations to human DNA. French microbiologist, geneticist and biochemist Emmanuelle Charpentier and American biochemist Jennifer A. Doudna, who shared the 2020 Nobel Prize in Chemistry for their work. — Popular Science, 17d ago
Evaluations of the roles that key agricultural biotechnology tools will play in the marketplace, such as next-generation DNA sequencing, biochips, RNA interference, and synthetic biology tools and genome editing tools,... — asdreports.com, 13d ago
Targeted protein degradation co-opts the cell’s natural disposal systems to remove disease-causing proteins and is applicable to diverse therapeutic areas including oncology, dermatology, immunology, neuroscience and respiratory diseases. This entirely new approach is revolutionising drug discovery by making the treatment of diseases previously thought to be undruggable a reality. — University of Dundee, 10d ago
Scientists used their expertise in quantum biology, artificial intelligence and bioengineering to improve how CRISPR Cas9 genome editing tools work on organisms like microbes that can be modified to produce renewable fuels and chemicals. — ScienceDaily, 22d ago
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Cradick expressed similar sentiments. He has worked with gene editing technologies like zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases, as well as CRISPR in both academia and industry throughout his career including at institutions like Georgia Tech and companies like CRISPR Therapeutics prior to joining Excision. — GEN - Genetic Engineering and Biotechnology News, 11w ago
By now you have probably heard of CRISPR, the gene-editing tool which enables researchers to replace and alter segments of DNA. Like genetic tailors, scientists have been experimenting with "snipping away" the genes that make mosquitoes malaria carriers, altering food crops to be more nutritious and delicious, and in recent years begun human trials to overcome some of the most challenging diseases and genetic disorders. The potential of CRISPR to improve our lives is so phenomenal that in 2020, researchers Jennifer Doudna and Emmanuelle Charpentier, who developed the most precise version of the tool named CRISPR-Cas9, were awarded the Nobel Prize in chemistry. — ScienceDaily, 9w ago
Base editing better than CRISPR-Cas9 for gene editing in SCD: Study... — Sickle Cell Disease News, 10w ago