Tag: pegRNAs
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
