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Corrigendum: Enhancing AAV-microdystrophin gene therapy after repeat dosing by blocking phagocytosis

Rita Spathis1, Deeva Robles Kuriplach1, Sabrina Narvesen1

  • 1Department of Pharmaceutical Sciences, School of Pharmacy and Pharmaceutical Sciences, Binghamton University, Binghamton, NY, United States.

Frontiers in Immunology
|April 7, 2025
PubMed

Keywords:
AAV9DMDDuchenne muscular dystrophyTLRcomplementgene therapyimmune responsemdx

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Gene Therapy00:59

Gene Therapy

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 inserted. The...