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Sam C. Towne

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Open access Sep 2026

Systemic responses to intravenous rAAV administration in boys with Duchenne muscular dystrophy

Systemic gene therapies utilizing recombinant adeno-associated virus (rAAV) are in the clinic for the treatment of rare monogenic diseases. This includes delandistrogene moxeparvovec for Duchenne muscular dystrophy (DMD), which uses AAVrh74 vector to deliver a micro-dystrophin transgene to striated muscle. While th...

Brooke Harris, Brooke M. Seldin, Sam C. Towne et al. · 0 citations

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