Article

Dual AAV gene therapy using laminin-linking proteins ameliorates muscle and nerve defects in LAMA2-related muscular dystrophy

2025-09-17

Abstract excerpt

<h4>ABSTRACT</h4> Adeno-associated virus (AAV)-mediated gene replacement holds promise for treating genetic diseases but faces challenges due to AAV’s limited packaging capacity and potential immune responses to transgene products, especially in patients lacking endogenous protein. LAMA2-related muscular dystrophy (LAMA2 MD), a severe congenital disorder caused by loss of laminin-α2, presents both hurdles: the L...

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Literature Corpus work: ac69be9b-fc1b-5fef-a26e-6aef05e520d2
DOI: 10.1101/2025.09.16.676550

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