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Development of viral vectors with optimal transgene expression for ocular gene therapies

Takao Hashimoto1

  • 1Jules Stein Eye Institute, University of California Los Angeles, CA 90095, U.S.A. gbf00573@nifty.com

Advances in Experimental Medicine and Biology
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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...