sábado, 9 de diciembre de 2023

CRISPR 2.0: a new wave of gene editors heads for clinical trials Landmark approval of the first CRISPR therapy paves the way for treatments based on more efficient and more precise genome editors.

https://www.nature.com/articles/d41586-023-03797-7 From the article: "Less than a month after the world’s first approval of a CRISPR-Cas9 genome-editing therapy, researchers are hoping that the therapy will win its second authorization this week — this time from the United States, with its famously stringent regulators and lucrative health-care market. The therapy, which UK regulators approved on 16 November, disables a gene as a means of treating a genetic blood disorder called sickle cell disease. A host of other CRISPR-Cas9 therapies that work on the same principle are in clinical trials as treatments for a range of diseases. " In historic decision, FDA approves a CRISPR-based medicine for treatment of sickle cell disease Adam Feuerstein By Adam Feuerstein Dec. 8, 2023 https://www.statnews.com/2023/12/08/fda-approves-casgevy-crispr-based-medicine-for-treatment-of-sickle-cell-disease/?utm_campaign=breaking_news&utm_medium=email&_hsmi=285761583&_hsenc=p2ANqtz-_V7DwrXJltdrMze1znber5DaiONrebb3FmkQ3cdH99-h23qHgHabK1WA-3mhzo6rs_Ea01ZfCX_OZcNXfaDM3ZF2usSg&utm_content=285761583&utm_source=hs_email From the article: "The Food and Drug Administration on Friday approved the world’s first medicine based on CRISPR gene-editing technology, a groundbreaking treatment for sickle cell disease that delivers a potential cure for people born with the chronic and life-shortening blood disorder. The new medicine, called Casgevy,'s authorization is a scientific triumph for the technology that can efficiently and precisely repair DNA mutations — ushering in a new era of genetic medicines for inherited diseases. "

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