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AAV gene therapy for Duchenne Muscular Dystrophy: lessons learned from a phase 3 trial

Giovanni Baranello1, Francesco Muntoni2,3

  • 1The Dubowitz Neuromuscular Centre, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, NIHR Great Ormond Street Hospital Biomedical Research Centre & Great Ormond Street Hospital NHS Foundation Trust, 30 Guilford Street, London, UK. g.baranello@ucl.ac.uk.

Gene Therapy
|October 24, 2024
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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