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When pullulanase needs a little push: MyoAAV capsids enhance gene therapy for GSD IIIa

Sree Venigalla1, Christina A Pacak1,2,3

  • 1Greg Marzolf Jr. Muscular Dystrophy Center and Department of Neurology, University of Minnesota Medical School, Minneapolis, MN, USA.

Molecular Therapy. Methods & Clinical Development
|December 17, 2025
PubMed
Abstract

No abstract available in PubMed .

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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