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Enhancing AAV-microdystrophin gene therapy after repeat dosing by blocking phagocytosis

BackgroundInefficient transduction is a major limitation in achieving therapeutic levels of AAV-delivered microdystrophin capable of improving muscle function in patients with Duchenne muscular dystrophy. Additionally, some patients experience acute complications due to activation of innate immune p...

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Principais autores: Rita Spathis, Deeva Robles Kuriplach, Sabrina Narvesen, Matthew Eybs, Karen Huang, Steven Torres, Madison King, Elizabeth Bagley, Pia Elustondo, Michael W. Lawlor, Kanneboyina Nagaraju, Melissa Morales
Formato: Artigo
Idioma:Inglês
Publicado em: Frontiers Media S.A. 2025-03-01
coleção:Frontiers in Immunology
Assuntos:
AAV9
mdx
Duchenne muscular dystrophy
DMD
gene therapy
immune response
Acesso em linha:https://www.frontiersin.org/articles/10.3389/fimmu.2025.1527840/full
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