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Switching on the lights: the use of optogenetics to advance retinal gene therapy

Therese Cronin1, Jean Bennett

  • 1FM Kirby Center for Molecular Ophthalmology, University of Pennsylvania, Philadelphia, Pennsylvania, USA. theresec@mail.med.upenn.edu

Molecular Therapy : the Journal of the American Society of Gene Therapy
|July 2, 2011
PubMed
Abstract

No abstract available in 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...