Tag: pegRNAs

The Next Chapter in Genome Editing? Tome Emerges from Stealth with $213M

Rahul Kakkar, MD, Tome Biosciences president and CEO While the CRISPR community celebrates the recent first FDA approval of a CRISPR-Cas9 therapy (Casgevy), new entities are making strides in their efforts to write perhaps the definitive new chapter in programmable genome editing. Last week, Tome Biosciences—a developer of genome editing…

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Prime Editing Comes of Age

CRISPR is perhaps the most popular tool in the genome editing toolkit. However, despite CRISPR’s obvious success, there are concerns about double-stranded breaks leading to unwanted, potentially dangerous edits.1 At the same time, some applications might require larger changes than the fairly limited single-base substitution repertoire of base editors. Prime…

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Gene Editing Tools Based on CRISPR/Cas System and Their Improvement Strategies

     Gene Editing Tools Based on CRISPR/Cas System and Their Improvement Strategies XIN Yuan1,TIAN Kai-ren3,QIAO Jian-jun1,2,3,CAIYIN Qing-ge-le1,2,**() 1 School of…

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Postdoctoral Researcher in Plant Biotechnology and Genome Editing

Category Research / Academic Location Amsterdam Crispr/Cas technology has revolutionized plant biotechnology by making targeted genetic modifications possible. Are you fascinated by the seemingly unlimited potential of this technology, and do you want to contribute to its further development and apply it to broaden plant immunity? Then this may be…

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CRISPR prime editing for unconstrained correction of oncogenic KRAS variants

Design of universal pegRNAs for KRAS gene correction We first investigated the mutation frequency of three RAS family genes, HRAS, NRAS, and KRAS, which are involved in tumorigenesis. According to the COSMIC (Catalog Of Somatic Mutations In Cancer) database (cancer.sanger.ac.uk/)22, KRAS mutations account for the majority (81.4%) of RAS mutations,…

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David Liu Hails Rapid Progress in Precision Genome Editing

David Liu takes center stage at ASGCT. [Kevin Davies] LOS ANGELES – David Liu, PhD, the Harvard University and Broad Institute chemist leading the development of base and prime editing technologies, began his American Society of Gene and Cell Therapy (ASGCT) presidential symposium keynote in humorous vein. That morning, Liu…

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CRISPR-Based Prime Editing Generates Mouse Models of Cancer Mutations

Genomic studies of cancer patients have revealed thousands of mutations linked to tumor development, but researchers are unsure how the vast majority of these mutations contribute to cancer because there’s no easy way to study them in animal models. Massachusetts Institute of Technology researchers have now developed a way to…

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Precise genome editing approach can correct mutation that causes sickle cell disease

Improving precision gene editing “We have identified what might be the next wave of therapies for genetic anemias,” said co-author Mitchell Weiss, M.D., Ph.D., St. Jude Department of Hematology chair. “We took the newest cutting-edge genetic engineering technology and showed that we could make meaningful gene edits for future therapies.” While the…

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Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice

Optimizing prime editing systems for HSPCs We previously reported the use of prime editing to correct HBBS by plasmid transfection in HEK293T cells containing the SCD mutation, reaching up to 58% efficiency (Fig. 1a)24. In contrast to HEK293T cells, HSPCs are difficult to transfect with plasmid DNA but are amenable…

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Genome Editing Tool Looks Beyond CRISPR and Prime

Omar Abudayyeh, PhD, McGovern fellow at MIT’s McGovern Institute for Brain Research A recently patented genome editing tool called PASTE holds genuine promise for expanding the universe of treatable genetic diseases. The approach combines elements of CRISPR and prime editing with a pair of enzymes designed to enable the integration…

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Recent advances in CRISPR-based genome editing technology and its applications in cardiovascular research | Military Medical Research

Doudna JA, Charpentier E. Genome editing. The new frontier of genome engineering with CRISPR-Cas9. Science. 2014;346(6213):1258096. Article  PubMed  Google Scholar  Doudna JA. The promise and challenge of therapeutic genome editing. Nature. 2020;578(7794):229–36. Article  CAS  PubMed  PubMed Central  Google Scholar  Nambiar TS, Baudrier L, Billon P, Ciccia A. CRISPR-based genome editing…

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Researchers develop a new tool using AI to boost genome editing

Researchers at the University of Zurich have developed a new tool that uses artificial intelligence to predict the efficacy of various genome-editing repair options. Unintentional errors in the correction of DNA mutations of genetic diseases can thus be reduced. Genome editing technologies offer great opportunities for treating genetic diseases. Methods…

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Artificial intelligence improves efficiency o

Genome editing technologies offer great opportunities for treating genetic diseases. Methods such as the widely used CRISPR/Cas9 gene scissors directly address the cause of the disease in the DNA. The scissors are used in the laboratory to make targeted modifications to the genetic material in cell lines and model organisms…

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Artificial intelligence improves efficiency of genome editing

Feb 02, 2023 (Nanowerk News) Researchers at the University of Zurich have developed a new tool that uses artificial intelligence to predict the efficacy of various genome-editing repair options. Unintentional errors in the correction of DNA mutations of genetic diseases can thus be reduced. Genome editing technologies offer great opportunities…

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Programmable CRISPR-Based Tool ‘Pastes’ in New Genes While Avoiding Double-Strand Breaks

NEW YORK — Researchers at MIT have developed a programmable tool that combines CRISPR and integrases to cut out unwanted stretches of DNA and paste in a replacement. While CRISPR-Cas9 gene editing can make such swaps, it is limited by the need for double-stranded DNA breaks, which can induce undesired…

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Enhancing Prime Editing Efficiency and Flexibility with Tethered and Split pegRNAs

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CRISPR-Cas9 Gene Therapy for Duchenne Muscular Dystrophy

Ishino Y, Shinagawa H, Makino K, et al. Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli, and identification of the gene product. J Bacteriol. 1987;169:5429–33. CAS  PubMed  PubMed Central  Google Scholar  Jansen R, van Embden JDA, Gaastra W, et al. Identification of genes…

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Twin Prime Editing Promises More Precise DNA Changes Without Double-Strand Breaks

NEW YORK – Researchers at the Broad Institute have developed a new prime editing method that allows for more precise replacement or excision of DNA sequences at endogenous human genomic sites, without the need for double-strand DNA breaks (DSBs). In a paper published on Thursday in Nature Biotechnology, researchers led…

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