AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis

por Buj-Bello, Anna, Fougerousse, Françoise, Schwab, Yannick, Messaddeq, Nadia, Spehner, Danièle, Pierson, Christopher R., Durand, Muriel, Kretz, Christine, Danos, Olivier, Douar, Anne-Marie, Beggs, Alan H., Schultz, Patrick, Montus, Marie, Denèfle, Patrice, Mandel, Jean-Louis
Publicado 2008

Myotubular myopathy and the neuromuscular junction: a novel therapeutic approach from mouse models

por Dowling, James J., Joubert, Romain, Low, Sean E., Durban, Ashley N., Messaddeq, Nadia, Li, Xingli, Dulin-Smith, Ashley N., Snyder, Andrew D., Marshall, Morgan L., Marshall, Jordan T., Beggs, Alan H., Buj-Bello, Anna, Pierson, Christopher R.
Publicado 2012