Translational and Regulatory Challenges for Exon Skipping Therapies

Aartsma-Rus, A and Ferlini, A and Goemans, N and Pasmooij, A M G and Wells, D J and Bushby, K and Vroom, E and Balabanov, P (2014) Translational and Regulatory Challenges for Exon Skipping Therapies. HUMAN GENE THERAPY, 25 (10). pp. 885-892.

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