Gene therapy for ocular diseases
Gene therapy is the use of genetic material to treat, cure, or prevent a disease or medical condition. It involves the introduction of genetic material into a target cell. The most common gene therapy approaches include replacing missing or mutated genes with a healthy copy of the gene, inactivating (knocking out) a mutated improperly functioning gene, introducing a new counteracting gene to help fighting manifestations of a disease, and altering the regulation of a certain gene. The principle of gene therapy is based on the transfer of a therapeutic gene using viral (adenoviruses, adeno-associated viruses, lentiviruses, herpes viruses) or non-viral (naked DNA, oligonucleotides, cationic liposomes and polymers) vectors administered to the target cell. As a result, the patient's cells will start to produce proteins that will correct a genetic disorder or acquired disease.Gene therapy is a very active area of research. In less than 20 years, it has made significant medical advances, from the concept to technology development and from experimental research to clinical translational studies. Proof-of-concept for gene therapies established in experimental models of human ocular diseases paved the way for development of gene therapy approaches for a broad range of eye disorders. Pioneering gene replacement therapies in two genetic diseases, Leber congenital amaurosis and Stargardt disease, and in age-related macular degeneration are currently under way to evaluate the safety and tolerability of these treatments. The Centre Hospitalier National d'Ophthalmologie des Quinze-Vingts, Paris, France with a principal investigator Prof. José-Alain Sahel is one of the locations of the clinical study of StarGen in patients with Stargardt macular degeneration.
The table below (PDF) summarizes bibliographic data on major and recent gene therapy developments in experimental models of ocular diseases and ongoing clinical trials : Gene therapy for ocular diseases: experimental models of and clinical trials - Major and recent publications; non-exhaustive bibliographic study (2011)
Created on 08 December 2011. Last Updated on 19 June 2012.
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