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Safety and efficacy of gene therapy for RAG1-deficient SCID

Frank J T Staal1, Karin Pike-Overzet1, Sander de Kivit1

  • 1Leiden University Medical Center, Leiden, the Netherlands.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|March 19, 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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