Ontology highlight
ABSTRACT:
SUBMITTER: Dupont JB
PROVIDER: S-EPMC4445007 | biostudies-literature | 2015
REPOSITORIES: biostudies-literature
Dupont Jean-Baptiste JB Tournaire Benoit B Georger Christophe C Marolleau Béatrice B Jeanson-Leh Laurence L Ledevin Mireille M Lindenbaum Pierre P Lecomte Emilie E Cogné Benjamin B Dubreil Laurence L Larcher Thibaut T Gjata Bernard B Van Wittenberghe Laetitia L Le Guiner Caroline C Penaud-Budloo Magalie M Snyder Richard O RO Moullier Philippe P Léger Adrien A
Molecular therapy. Methods & clinical development 20150408
Preclinical gene therapy strategies using recombinant adeno-associated virus (AAV) vectors in animal models of Duchenne muscular dystrophy have shown dramatic phenotype improvements, but long-lasting efficacy remains questionable. It is believed that in dystrophic muscles, transgene persistence is hampered, notably by the progressive loss of therapeutic vector genomes resulting from muscle fibers degeneration. Intracellular metabolic perturbations resulting from dystrophin deficiency could also ...[more]