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Adeno-associated viral vectors for gene therapy of inherited retinal degenerations

John G Flannery1, Meike Visel

  • 1Neuroscience Division, Department of Molecular and Cell Biology, Helen Wills Neuroscience Institute, University of California, Berkeley, CA, USA. flannery@berkeley.edu

Methods in Molecular Biology (Clifton, N.J.)
|November 15, 2012
PubMed

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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...