A Year After Gene Therapy, Boys With Muscular Dystrophy Are Healthier and Stronger

Good News Notes:

Two and a half years ago, a study published in Science Advances detailed how the gene editing tool CRISPR/Cas-9 repaired genetic mutations related to Duchenne Muscular Dystrophy (DMD). The study was a proof of concept, and used induced pluripotent stem cells (iPSCs).

But now a similar treatment has not only been administered to real people, it has worked and made a difference in their quality of life and the progression of their disorder. Nine boys aged 6 to 12 who have been living with DMD since birth received a gene therapy treatment from pharmaceutical giant Pfizer, and a year later, 7 of the boys show significant improvement in muscle strength and function.

Though the treatment’s positive results are limited to a small group, they’re an important breakthrough for gene therapy, and encouraging not just for muscular dystrophy but for many other genetic diseases that could soon see similar treatments developed.”

View the whole story here: https://singularityhub.com/2020/07/30/a-year-after-getting-gene-therapy-boys-with-muscular-dystrophy-are-healthier-and-stronger/

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