Skip to main content
Journal cover image

A phase 1 study of gene therapy with ACTUS-101 in late-onset Pompe disease

Publication ,  Conference
Koeberl, D; Smith, EC; Case, LE; Hopkins, S; Childers, MK; Walters, C; Han, S-O; Spears, TG; Hornik, CP; Bali, D; Kishnani, PS
Published in: Molecular Genetics and Metabolism
February 2020

Duke Scholars

Published In

Molecular Genetics and Metabolism

DOI

ISSN

1096-7192

Publication Date

February 2020

Volume

129

Issue

2

Start / End Page

S91 / S91

Publisher

Elsevier BV

Related Subject Headings

  • Genetics & Heredity
  • 3202 Clinical sciences
  • 3105 Genetics
  • 1103 Clinical Sciences
 

Citation

APA
Chicago
ICMJE
MLA
NLM
Koeberl, D., Smith, E. C., Case, L. E., Hopkins, S., Childers, M. K., Walters, C., … Kishnani, P. S. (2020). A phase 1 study of gene therapy with ACTUS-101 in late-onset Pompe disease. In Molecular Genetics and Metabolism (Vol. 129, pp. S91–S91). Elsevier BV. https://doi.org/10.1016/j.ymgme.2019.11.227
Koeberl, Dwight, Edward C. Smith, Laura E. Case, Sam Hopkins, Martin K. Childers, Crista Walters, Sang-oh Han, et al. “A phase 1 study of gene therapy with ACTUS-101 in late-onset Pompe disease.” In Molecular Genetics and Metabolism, 129:S91–S91. Elsevier BV, 2020. https://doi.org/10.1016/j.ymgme.2019.11.227.
Koeberl D, Smith EC, Case LE, Hopkins S, Childers MK, Walters C, et al. A phase 1 study of gene therapy with ACTUS-101 in late-onset Pompe disease. In: Molecular Genetics and Metabolism. Elsevier BV; 2020. p. S91–S91.
Koeberl, Dwight, et al. “A phase 1 study of gene therapy with ACTUS-101 in late-onset Pompe disease.” Molecular Genetics and Metabolism, vol. 129, no. 2, Elsevier BV, 2020, pp. S91–S91. Crossref, doi:10.1016/j.ymgme.2019.11.227.
Koeberl D, Smith EC, Case LE, Hopkins S, Childers MK, Walters C, Han S-O, Spears TG, Hornik CP, Bali D, Kishnani PS. A phase 1 study of gene therapy with ACTUS-101 in late-onset Pompe disease. Molecular Genetics and Metabolism. Elsevier BV; 2020. p. S91–S91.
Journal cover image

Published In

Molecular Genetics and Metabolism

DOI

ISSN

1096-7192

Publication Date

February 2020

Volume

129

Issue

2

Start / End Page

S91 / S91

Publisher

Elsevier BV

Related Subject Headings

  • Genetics & Heredity
  • 3202 Clinical sciences
  • 3105 Genetics
  • 1103 Clinical Sciences