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W. Lostal

French Institute of Health and Medical Research

6

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P.156 - Preclinical studies for calpain 3 gene transfer: primate pilot study and dose effect evaluation

2017 Neuromuscular Disorders
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A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence.

2015 Molecular therapy. Methods & clinical development
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High expression and therapeutic efficacy of systemic delivery of a dual AAV strategy in a murine model for dysferlin deficiency

2015 Neuromuscular Disorders
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T.O.2 Efficient recovery of dysferlin deficiency by dual adeno associated vector mediated gene transfer

2009 Neuromuscular Disorders
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G.O.5 Partial functionality of a Mini-dysferlin molecule identified in a patient affected with moderately severe primary dysferlinopathy

2008 Neuromuscular Disorders
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