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Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem

Jacqueline Corrigan-Curay1, Odile Cohen-Haguenauer, Marina O'Reilly

  • 1Biotechnology Activities, National Institutes of Health, Bethesda, Maryland, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|June 2, 2012
PubMed
Abstract

No abstract available in PubMed .

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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...