Long‐Term Correction of Murine Glycogen Storage Disease Type III by AAV‐Mediated Gene Therapy Using an Immunotolerizing Dual Promoter to Express Bacterial Pullulanase
Our results demonstrated the long-term efficacy and safety of systemic AAV9-Dual-Pull delivery in GSD IIIa mice. Future studies will test this gene therapy approach in GSD IIIa dogs prior to the clinical translation to GSD III patients.
Authors
- Jeong‐A Lim (ORCID: https://orcid.org/0000-0002-4373-4800)
- Kuo An Liao (ORCID: https://orcid.org/0000-0002-4708-7479)
- Haiqing Yi (ORCID: https://orcid.org/0000-0003-2254-6226)
- Baodong Sun (ORCID: https://orcid.org/0000-0002-2191-0025)
- Su Jin Choi (ORCID: https://orcid.org/0000-0001-7681-6493)
Institutions
- Duke University (US)
Publication Details
- Journal
- Advances in Cell and Gene Therapy
- Published
- 2025-01-01
- DOI
- https://doi.org/10.1155/acg2/4639392
- Citations
- 1
- Primary Topic
- Glycogen Storage Diseases and Myoclonus
- Type
- article
- Field-Weighted Citation Impact
- 1.67
Funders
- Duke University
- National Institute of Arthritis and Musculoskeletal and Skin Diseases