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UW researchers will trial gene editing therapy to treat blindness

UW researchers will trial gene editing therapy to treat blindness
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A research partnership brings gene editing promise to eye disease - Morgridge Institute for Research

One of the first frontiers of CRISPR gene editing is targeting diseases related to the eye. A diverse Morgridge and UW-Madison team is working to make future applications precise, safe and reproducible.

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Genome editing 'toolkit' to speed up gene therapy development

Genome editing toolkit to speed up gene therapy development Appeared in BioNews 1090 A genome editing consortium funded by the US National Institutes of Health (NIH) has unveiled plans to develop a new genome editing toolkit to accelerate the development of gene therapies. The Somatic Cell Genome Editing Consortium (SCGE) was granted $190 million to develop standardised genome editing approaches and methods, the outline for which was published last week. The overall goal is to facilitate the development of genome editing approaches for the treatment of multiple diseases, by providing a standardised starting platform. The consortium encompasses 75 principal investigators across 38 institutions. NIH realised it was important for all of us who are investigating gene editing to work together toward a common goal, said author and SCGE member Professor Danith Ly of Carnegie Mellon University, Pennsylvania. What we ll end up with is a very valuable, rigorously evaluated resource

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Connecting enhancer variants, genes, and disease; synthetic spike binders; measuring tumors' cellular mix; and more

Posted April 9th, 2021 for Broad Institute Welcome to the April 9, 2021 installment of Research Roundup , a recurring snapshot of recent studies published by scientists at the Broad Institute and their collaborators. Enhancing V2F maps GWASs have yielded thousands of genetic variants linked to disease. To help figure out the function of many of these variants, Joseph Nasser (now at Caltech), Drew Bergman, Charles Fulco (now at Bristol Myers Squibb), Philine Guckelberger (now at the Free University of Berlin), Benjamin Doughty (now at Stanford University), Eric Lander (on leave), Jesse Engreitz (now at Stanford), and colleagues used their activity-by-contact (ABC) model to build maps that connect enhancers to their target genes in 131 cell types and tissues. Using those maps, the team linked more than 5,000 GWAS signals to nearly 2,250 genes across 72 traits and diseases. The researchers also predicted which enhancers contain risk variants for inflammatory bowel di

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Erik Sontheimer co-leading efforts to develop gene editing toolkit by NIH Somatic Cell Genome Editing Consortium

Erik Sontheimer co-leading efforts to develop gene editing toolkit by NIH Somatic Cell Genome Editing Consortium By Jim Fessenden April 07, 2021 Six UMass Medical School scientists are among the members of the National Institutes of Health’s Somatic Cell Genome Editing Consortium to publish a paper in Nature outlining the program’s goals to accelerate and benchmark the development of safer, more effective methods to edit the genomes of disease-relevant somatic cells in patients. Their research comprising new genome editors, delivery technologies, methods for tracking edited cells in vivo, animal models and human biological systems will be made available as a toolkit that other biomedical researchers can access and apply to an array of disease types and cell tissues as a means of accelerating the clinical development of new therapies for a wide range of illnesses.

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