title: Phoenix rising: gene therapy makes a comeback.
abstract: Despite the first application of gene therapy in 1990, gene therapy has until recently failed to meet the huge expectations set forth by researchers, clinicians, and patients, thus dampening enthusiasm for an imminent cure for many life-threatening genetic diseases. Nonetheless, in recent years we have witnessed a strong comeback for gene therapy, with clinical successes in young and adult subjects suffering from inherited forms of blindness or from X-linked severe combined immunodeficiency disease. In this review, various gene therapy vectors progressing into clinical development and pivotal advances in gene therapy trials will be discussed.
authors.affiliation: Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA 19104-3403, USA. limberis@mail.med.upenn.edu
authors.forename: Maria P
authors.initials: MP
authors.lastname: Limberis
mesh_terms: D000243:Adenosine Deaminase / Q000172:deficiency / Q000235:genetics; D000256:Adenoviridae / Q000378:metabolism; D000818:Animals; D000229:Dependovirus / Q000378:metabolism; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000378:metabolism; D006801:Humans; D016086:Lentivirus / Q000378:metabolism; D012190:Retroviridae / Q000378:metabolism; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000628:therapy; D013601:T-Lymphocytes / Q000166:cytology; D053632:X-Linked Combined Immunodeficiency Diseases / Q000235:genetics / Q000628:therapy
chemical_list: D000243:Adenosine Deaminase