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A roadmap to safe, efficient, and stable lentivirus-mediated gene therapy with hematopoietic cell transplantation

Anton Neschadim1, J Andrea McCart, Armand Keating

  • 1Division of Stem Cell and Developmental Biology, Ontario Cancer Institute, Toronto, Ontario, Canada.

Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation
|November 21, 2007
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...
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...