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- W1987472017 abstract "Gene therapy approaches for DMD using recombinant adeno-associated viral (rAAV) vectors have the potential to halt dystrophic progression and reverse aspects of the muscle pathophysiology. Advantages of rAAV-delivery of micro-dystrophins include long lasting (years) expression of the delivered gene cassette, potential for systemic delivery to all muscles of the body (including the heart) and applicability to all patients regardless of their mutation. However, AAV vectors have a limited cloning capacity of" @default.
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- W1987472017 date "2014-10-01" @default.
- W1987472017 modified "2023-09-26" @default.
- W1987472017 title "G.P.95" @default.
- W1987472017 doi "https://doi.org/10.1016/j.nmd.2014.06.109" @default.
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