Liver-Mediated Gene Therapy with an Engineered Secretable GAA Transgene Results in Whole-Body Correction of Pompe Disease
Publication
, Conference
Ronzitti, G; Colella, P; Puzzo, F; Biferi, MG; Bali, D; Paulk, NK; Vidal, P; Collaud, F; Sola, MS; Charles, S; Hardet, R; Leborgne, C ...
Published in: MOLECULAR THERAPY
May 1, 2017
Duke Scholars
Published In
MOLECULAR THERAPY
EISSN
1525-0024
ISSN
1525-0016
Publication Date
May 1, 2017
Volume
25
Issue
5
Start / End Page
244 / 244
Location
Washington, DC
Publisher
CELL PRESS
Conference Name
20th Annual Meeting of the American-Society-of-Gene-and-Cell-Therapy (ASGCT)
Related Subject Headings
- Biotechnology
- 3206 Medical biotechnology
- 3202 Clinical sciences
- 3105 Genetics
- 11 Medical and Health Sciences
- 10 Technology
- 06 Biological Sciences
Citation
APA
Chicago
ICMJE
MLA
NLM
Ronzitti, G., Colella, P., Puzzo, F., Biferi, M. G., Bali, D., Paulk, N. K., … Mingozzi, F. (2017). Liver-Mediated Gene Therapy with an Engineered Secretable GAA Transgene Results in Whole-Body Correction of Pompe Disease. In MOLECULAR THERAPY (Vol. 25, pp. 244–244). Washington, DC: CELL PRESS.
Ronzitti, Giuseppe, Pasqualina Colella, Francesco Puzzo, Maria Grazia Biferi, Deeksha Bali, Nicole K. Paulk, Patrice Vidal, et al. “Liver-Mediated Gene Therapy with an Engineered Secretable GAA Transgene Results in Whole-Body Correction of Pompe Disease.” In MOLECULAR THERAPY, 25:244–244. CELL PRESS, 2017.
Ronzitti G, Colella P, Puzzo F, Biferi MG, Bali D, Paulk NK, et al. Liver-Mediated Gene Therapy with an Engineered Secretable GAA Transgene Results in Whole-Body Correction of Pompe Disease. In: MOLECULAR THERAPY. CELL PRESS; 2017. p. 244–244.
Ronzitti, Giuseppe, et al. “Liver-Mediated Gene Therapy with an Engineered Secretable GAA Transgene Results in Whole-Body Correction of Pompe Disease.” MOLECULAR THERAPY, vol. 25, no. 5, CELL PRESS, 2017, pp. 244–244.
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. Liver-Mediated Gene Therapy with an Engineered Secretable GAA Transgene Results in Whole-Body Correction of Pompe Disease. MOLECULAR THERAPY. CELL PRESS; 2017. p. 244–244.
Published In
MOLECULAR THERAPY
EISSN
1525-0024
ISSN
1525-0016
Publication Date
May 1, 2017
Volume
25
Issue
5
Start / End Page
244 / 244
Location
Washington, DC
Publisher
CELL PRESS
Conference Name
20th Annual Meeting of the American-Society-of-Gene-and-Cell-Therapy (ASGCT)
Related Subject Headings
- Biotechnology
- 3206 Medical biotechnology
- 3202 Clinical sciences
- 3105 Genetics
- 11 Medical and Health Sciences
- 10 Technology
- 06 Biological Sciences