The yin and yang of stem cell gene therapy: insights into hematopoiesis, leukemogenesis, and gene therapy safety.
Over the past decade, success in the treatment of serious genetic disorders via gene therapy was finally achieved. However, this progress was tempered by the occurrence of serious adverse events related to vector integration into the genome and activation of adjacent proto-oncogenes. Investigators are now focused on retaining the clinical potential of integrating vectors while decreasing the risk of insertional mutagenesis.
Dunbar
Cynthia E
CE
Hematology Branch, NIH, Building 10, Room 7C103, 9000 Rockville Pike, Bethesda, MD 20892, USA. dunbarc@nhlbi.nih.gov
D015316:Genetic Therapy / Q000009:adverse effects; D005822:Genetic Vectors; D006410:Hematopoiesis / Q000235:genetics*; D006801:Humans; D007938:Leukemia / Q000209:etiology*; D012190:Retroviridae / Q000235:genetics; D016662:Virus Integration