Skip to main content
Journal cover image

Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector.

Publication ,  Journal Article
Sun, B-D; Chen, Y-T; Bird, A; Amalfitano, A; Koeberl, DD
Published in: Mol Ther
February 2003

We administered an adenovirus-adeno-associated virus (Ad-AAV) vector encoding human acid alpha-glucosidase (hGAA) to acid alpha-glucosidase-knockout (GAA-KO) mice on day 3 of life by gastrocnemius injection. In contrast to previous results for muscle-targeted Ad vector in adult GAA-KO mice, the muscles of the hindlimb showed reduced glycogen content and persistent hGAA for as long as 6 months after neonatal Ad-AAV vector administration. Not only the injected gastrocnemius muscles, but also the hamstrings and quadriceps muscles produced therapeutic levels of hGAA as a result of widespread transduction with the Ad-AAV vector; moreover, hGAA activity was 50-fold elevated as compared to normal mice. Vector RNA was detected in the hindlimb muscles, the hearts, and the livers by northern blot analysis and/or by RT-PCR for as long as 6 months. The low levels of hGAA detected in the heart were attributable to transduction with the Ad-AAV vector, not to secretion of hGAA by the injected muscle and uptake by the heart. Finally, although an antibody response to hGAA was present, it did not prevent the correction of glycogen storage in the skeletal muscle of GAA-KO mice.

Duke Scholars

Altmetric Attention Stats
Dimensions Citation Stats

Published In

Mol Ther

DOI

ISSN

1525-0016

Publication Date

February 2003

Volume

7

Issue

2

Start / End Page

193 / 201

Location

United States

Related Subject Headings

  • Tissue Distribution
  • Time Factors
  • Reverse Transcriptase Polymerase Chain Reaction
  • Plasmids
  • Myocardium
  • Muscle, Skeletal
  • Mice, Knockout
  • Mice
  • Liver
  • Humans
 

Citation

APA
Chicago
ICMJE
MLA
NLM
Sun, B.-D., Chen, Y.-T., Bird, A., Amalfitano, A., & Koeberl, D. D. (2003). Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector. Mol Ther, 7(2), 193–201. https://doi.org/10.1016/s1525-0016(02)00055-2
Sun, Bao-dong, Y. -. T. Chen, Andrew Bird, Andrea Amalfitano, and Dwight D. Koeberl. “Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector.Mol Ther 7, no. 2 (February 2003): 193–201. https://doi.org/10.1016/s1525-0016(02)00055-2.
Sun B-D, Chen Y-T, Bird A, Amalfitano A, Koeberl DD. Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector. Mol Ther. 2003 Feb;7(2):193–201.
Sun, Bao-dong, et al. “Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector.Mol Ther, vol. 7, no. 2, Feb. 2003, pp. 193–201. Pubmed, doi:10.1016/s1525-0016(02)00055-2.
Sun B-D, Chen Y-T, Bird A, Amalfitano A, Koeberl DD. Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector. Mol Ther. 2003 Feb;7(2):193–201.
Journal cover image

Published In

Mol Ther

DOI

ISSN

1525-0016

Publication Date

February 2003

Volume

7

Issue

2

Start / End Page

193 / 201

Location

United States

Related Subject Headings

  • Tissue Distribution
  • Time Factors
  • Reverse Transcriptase Polymerase Chain Reaction
  • Plasmids
  • Myocardium
  • Muscle, Skeletal
  • Mice, Knockout
  • Mice
  • Liver
  • Humans