CRISPR/Cas9

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Novel gene-editing method improves vision in blind rats

December 6, 2016 At a Glance Scientists developed a targeted gene-replacement technique that can modify genes in both dividing and non-dividing cells in living animals. The method enabled replacement of a faulty gene in neurons and partially restored vision in blind rats. The technique thus holds promise as a potential tool for gene therapy. The

Sickle Cell Disease: Gene-Editing Tools Point to Possible Ultimate Cure

Posted on October 25, 2016 by Dr. Francis Collins Caption: An electron micrograph showing two red blood cells, one normal (right) and the other (left) deformed by crystalline hemoglobin into the “sickle” shape characteristic of patients with sickle cell disease. Credit: Frans Kuypers: RBClab.com, UCSF Benioff Children’s Hospital Oakland Scientists first described the sickle-shaped red

New gene editing method shows promising results for correcting muscular dystrophy

Medical XPress, Provided by UT Southwestern Medical Center ~ August 14, 2014 Researchers used a technique called CRISPR/Cas9-mediated genome editing, which can precisely remove a mutation in DNA, allowing the body's DNA repair mechanisms to replace it with a normal copy of the gene. The benefit of this over other gene therapy techniques is that