Whole-body correction of Pompe disease by AAV liver-mediated gene transfer of engineered secretable GAA transgenes

Conference Paper

Full Text

Duke Authors

Cited Authors

  • Puzzo, F; Colella, P; Biferi, MG; Bali, D; Paulk, NK; Vidal, P; Collaud, F; Simon-Sola, M; Charles, S; Hardet, R; Leborgne, C; Meliani, A; Cohen-Tannoudji, M; Astord, S; Gjata, B; Sellier, P; van Wittenberghe, L; Vignaud, A; Boisgerault, F; Barkats, M; Laforet, P; Kay, MA; Koeberl, DD; Ronzitti, G; Mingozzi, F

Published Date

  • December 1, 2017

Published In

Volume / Issue

  • 28 / 12

Start / End Page

  • A12 - A12

Published By


  • 1

Electronic International Standard Serial Number (EISSN)

  • 1557-7422

International Standard Serial Number (ISSN)

  • 1043-0342

Conference Name

  • European-Society-of-Gene-and-Cell-Therapy (ESCGT) Congress

Conference Location

  • Berlin, GERMANY

Conference Start Date

  • 2017