title: Gene therapy: concepts and methods. Few applications so far.
abstract: (1) Gene therapy consists of inserting foreign genetic material into a patient's cells to correct a health disorder; (2) Only a fraction of gene therapy trials involve hereditary single-gene disorders. Most trials focus on acquired disorders such as cancer and cardiovascular disease; (3) The therapeutic gene can be delivered directly into the target organ or the general circulation, or alternatively into cells that are first isolated from the patient and then reinjected; (4) One of the main problems with gene therapy is finding an appropriate vector (viral or non-viral) that is both effective and safe. Most currently available vectors confer only weak and transient therapeutic gene expression. In addition, many carry major risks such as immunological disorders and oncogenicity; (5) In early 2009, only one therapeutic gene product is commercially available, in China.
mesh_terms: D000818:Animals; D002318:Cardiovascular Diseases / Q000235:genetics / Q000628:therapy; D002681:China; D002986:Clinical Trials as Topic; D003550:Cystic Fibrosis / Q000235:genetics / Q000628:therapy; D018014:Gene Transfer Techniques; D030342:Genetic Diseases, Inborn / Q000235:genetics / Q000628:therapy*; D015316:Genetic Therapy / Q000009:adverse effects / Q000379:methods*; D005822:Genetic Vectors; D002836:Hemophilia B / Q000235:genetics / Q000628:therapy; D006801:Humans; D007239:Infections / Q000235:genetics / Q000628:therapy; D009369:Neoplasms / Q000235:genetics / Q000628:therapy; D020163:Ornithine Carbamoyltransferase Deficiency Disease / Q000235:genetics / Q000628:therapy; D019076:Transgenes / Q000235:genetics*; D053632:X-Linked Combined Immunodeficiency Diseases / Q000235:genetics / Q000628:therapy