Severe side effects in gene therapy for muscular dystrophy

Experimental treatment for Duchenne muscular dystrophy started in December 2017 under NCT03368742 assignation. Clinicians attempted to insert adeno-associated virus (AAV9), which would establish muscle-specific expression of microdystrophin. They planned to enroll 16 individuals, including control group.

Sixth patient in the second (higher) dosing cohort experienced severe side effects, typically associated with immunological response to the viral load:

thrombocytopenia, a decrease in red blood cell count, acute kidney injury, and cardio-pulmonary insufficiency


Spokesperson for the company assured that the patient is recovering and scientists are fully cooperating with FDA to diagnose the cause and introduce corrections.

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