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| Q37880689 | Organ targeted prenatal gene therapy--how far are we? |
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| Q34870765 | Recent advances in inducible expression in transgenic mice |
| Q35904461 | Recombinant adeno-associated viral (rAAV) vectors as therapeutic tools for Duchenne muscular dystrophy (DMD). |
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| Q45033314 | Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study |
| Q58591574 | Satellite cells maintain regenerative capacity but fail to repair disease-associated muscle damage in mice with Pompe disease |
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