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


Buj-Bello, A., Fougerousse, F., Schwab, Y., Messaddeq, N., Spehner, D., Pierson, C.R., Durand, M., Kretz, C., Danos, O., Douar, A.M., Beggs, A.H., Schultz, P., Montus, M., Denefle, P. and Mandel, J.L.:

Hum Mol Genet 17 (2008) 2132-2143.

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