gene

Replay targets genetic brain disorders with new gene therapy company, Kaleibe

Replay targets genetic brain disorders with new gene therapy company, Kaleibe

Replay, a genome-writing company, today announced the launch of Kaleibe, an HSV gene therapy company targeting genetic brain disorders. This is the third of Replay’s product companies to launch since the company’s inception in July and will leverage Replay’s high-carrying herpes simplex virus (HSV) delivery vector, synHSV, to target genetic disorders of the brain. Kaleibe …

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Promising study suggests AAV-based gene therapy can treat type I glutaric aciduria

Promising study suggests AAV-based gene therapy can treat type I glutaric aciduria

Treatment of glutaric aciduria type I (GA-I) by intracerebroventricular administration of GCDH. Credit: Institute of Zoology, Chinese Academy of Sciences, China Glutaric aciduria type I (GA-I) is an inherited disease caused by a mutation in the glutaryl-CoA dehydrogenase (GCDH) gene. The global incidence of GA-I is estimated at 1/100,000, with approximately 75,000 cases worldwide. Patients …

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Discovery of a new function of the CRISPR gene scissors: the protein scissors activate the defense function

Discovery of a new function of the CRISPR gene scissors: the protein scissors activate the defense function

Uncropped and unedited version of all gels used in this study. The red box indicates the portion of the gel that is shown in the indicated figures. Credit: Nature (2022). DOI: 10.1038/s41586-022-05571-7 For several years, the CRISPR/Cas9 gene scissors have caused a stir in science and medicine. This new molecular biology tool has its origins …

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