Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes

Autor:	Nicole K. Paulk, Marcelo Simon-Sola, Dwight D. Koeberl, Mark A. Kay, Fanny Collaud, M. Biferi, Florence Boisgerault, Francesco Puzzo, Patrice Vidal, Deeksha Bali, Christian Leborgne, Federico Mingozzi, Pascal Laforêt, Pauline Sellier, Martine Barkats, Severine Charles, Romain Hardet, L. Van Wittenberghe, Pasqualina Colella, Giuseppe Ronzitti
Rok vydání:	2017
Předmět:	0301 basic medicine business.industry Transgene Disease Virology 03 medical and health sciences 030104 developmental biology Neurology Pediatrics Perinatology and Child Health Medicine Neurology (clinical) Whole body business Genetics (clinical)
Zdroj:	Neuromuscular Disorders. 27:S246
ISSN:	0960-8966
DOI:	10.1016/j.nmd.2017.06.543
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::52475560f59b5dc256a75a4d37e3a5fd https://doi.org/10.1016/j.nmd.2017.06.543 Zobrazit plný text záznamu Full Text from ScienceDirect