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Author

Brooke Harris

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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
Open access Sep 2026

Mechanisms of Cardiac Cell Death in Duchenne Muscular Dystrophy

Results suggest that apoptotic and pyroptotic pathways are not active in these tissue samples, suggesting that alternative cell death pathways, such as necroptosis, may be responsible, or these animals had not undergone significant cardiomyocyte cell death.

Caroline Lee, Ryan W. Krueger, Brooke Harris et al. · 0 citations

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