title: Gene therapy: the promise of a permanent cure.
abstract: Gene therapy offers the possibility of a permanent cure for any of the more than 10,000 human diseases caused by a defect in a single gene. Among these diseases, the hemophilias represent an ideal target, and studies in both animals and humans have provided evidence that a permanent cure for hemophilia is within reach.
authors: Wake Forest Institute for Regenerative Medicine, 391 Technology Way, Winston-Salem, NC 27157, USA. cporada@wakehealth.edu.
authors: Christopher D
authors: CD
authors: Porada
authors: Christopher
authors: C
authors: Stem
authors: Graca
authors: G
authors: Almeida-Porada
mesh_terms: D015316:Genetic Therapy; D006467:Hemophilia A / Q000235:genetics* / Q000628:therapy*; D006801:Humans