Liver-Mediated Gene Therapy with an Engineered Secretable GAA Transgene Results in Whole-Body Correction of Pompe Disease


Conference Paper

Full Text

Duke Authors

Cited Authors

  • Ronzitti, G; Colella, P; Puzzo, F; Biferi, MG; Bali, D; Paulk, NK; Vidal, P; Collaud, F; Sola, MS; 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; Mingozzi, F

Published Date

  • May 1, 2017

Published In

Volume / Issue

  • 25 / 5

Start / End Page

  • 244 - 244

Published By


  • 1

Electronic International Standard Serial Number (EISSN)

  • 1525-0024

International Standard Serial Number (ISSN)

  • 1525-0016

Conference Name

  • 20th Annual Meeting of the American-Society-of-Gene-and-Cell-Therapy (ASGCT)

Conference Location

  • Washington, DC

Conference Start Date

  • May 10, 2017

Conference End Date

  • May 13, 2017