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Reduced-intensity conditioning in LV-mediated gene therapy for Fabry disease targeting HSPCs

Aneal Khan1, Dwayne L Barber2, Ronan Foley3

  • 1M.A.G.I.C. (Metabolics and Genetics in Canada) Clinic, Calgary, AB, Canada.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|August 14, 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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