Molecular genetics and metabolism
Novel promoters drive therapeutic transgene expression and evade transgene-specific immune responses in a mouse model of Pompe disease.
Baodong Sun, Haiqing Yi, Sang-Oh Han, Songtao Li, William Eisner, Elizabeth D Brooks, Rebecca Bacon, Dwight D Koeberl
Published: 202510.1016/j.ymgme.2025.109717
Abstract
Systemically administered gene therapy is under development for the treatment of Pompe disease, an inherited lysosomal storage disorder caused by acid alpha-glucosidase (GAA) deficiency. We evaluated recombinant AAV9 vectors expressing GAA driven by…
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