Exon Skipping as a Therapeutic Strategy Applied to an...

Human Gene Therapy 2013 / 07 Vol. 24; Iss. 7

Exon Skipping as a Therapeutic Strategy Applied to an RYR1 Mutation with Pseudo-Exon Inclusion Causing a Severe Core Myopathy

Rendu, John, Brocard, Julie, Denarier, Eric, Monnier, Nicole, Piétri-Rouxel, France, Beley, Cyriaque, Roux-Buisson, Nathalie, Gilbert-Dussardier, Brigitte, Perez, Marie José, Romero, Norma, Garcia, Lu
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Volume:
24
Language:
english
Journal:
Human Gene Therapy
DOI:
10.1089/hum.2013.052
Date:
July, 2013
File:
PDF, 533 KB
english, 2013
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