Myotonic dystrophy type 1 (DM1)

article

How engineered RNA binding proteins delivered in vivo by gene therapy could treat myotonic dystrophy

13 July 2021 | By Dr Jim Burns (Locanabio)

In this article, Dr Jim Burns discusses promising pre-clinical results of how a new platform could treat the root cause of many devastating genetic diseases including myotonic dystrophy type 1.

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Myotonic dystrophy type 1 (DM1)

How engineered RNA binding proteins delivered in vivo by gene therapy could treat myotonic dystrophy