Progress towards gene therapy

Progress towards gene therapy for haemophilia B.

Haemophilia B is an X-linked recessive bleeding disorder, arising from a deficiency of coagulation factor IX. It has been a target for gene therapy ever since the factor IX gene was cloned in 1982. Several distinct approaches have been evaluated in humans over the last 30 years, but none has resulted in tangible corrections of the bleeding phenotype in humans until recently. Our group has now s...

Gene therapy: progress and predictions

The first clinical gene delivery, which involved insertion of a marker gene into lymphocytes from cancer patients, was published 25 years ago. In this review, we describe progress since then in gene therapy. Patients with some inherited single-gene defects can now be treated with their own bone marrow stem cells that have been engineered with a viral vector carrying the missing gene. Patients w...

Progress toward human gene therapy.

Gene Therapy: Progress and Challenges

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 ri...

Gene Therapy 2017: Progress and Future Directions

Gene therapy has changed dramatically in the 28 years since the first human gene transfer experiment in 1989. Alipogene tiparvovec, GlyberaR R ©, a recombinant adenoassociated virus (rAAV) product for lipoprotein lipase deficiency, and Strimvelis R ©, a lentivirus vector for severe combined immune deficiency are approved in Europe. An rAAV2 product for a congenital form of blindness is currentl...