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.
作者:Cynthia E, Dunbar
来源:Hematology. American Society of Hematology. Education Program 2007 年