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Astellas' gene therapy takes another hit

 Last Update: 2022-05-22
 Source: Internet
 Author: User

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Duchenne muscular dystrophy (DMD) is the most common type of progressive muscular dystrophy, with progressive and lethal features

Duchenne muscular dystrophy (DMD) is caused by the absence or loss of function of dystrophin

The gene encoding dystrophin is located at Xp21.

Japanese pharmaceutical company Astellas has disclosed that it will discontinue its AT702, AT751 and AT753 gene therapy programs for Duchenne muscular dystrophy (DMD) based on recent preclinical study data

The company's gene therapy approach for DMD uses an AAV vector encoding a modified U7 snRNA to deliver an antisense sequence designed to induce cells to skip the erroneous or misplaced genetic code in the dystrophin gene, thereby restoring functional dystrophin Production of atrophin

Original source:

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