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Recombinant adeno-associated virus as delivery vector for gene therapy--a review

Yang Lu1

  • 1Department of Neurology, The Johns Hopkins University School of Medicine, Baltimore, MD 21287, USA. ylu@jhmi.edu

Stem Cells and Development
|April 8, 2004
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...