title: Gene therapy: progress and predictions.
abstract: 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 with inherited retinopathies and haemophilia B can also be treated by local or systemic injection of viral vectors. There are also a number of promising gene therapy approaches for cancer and infectious disease. We predict that the next 25 years will see improvements in safety, efficacy and manufacture of gene delivery vectors and introduction of gene-editing technologies to the clinic. Gene delivery may also prove a cost-effective method for the delivery of biological medicines.
authors.affiliation: Division of Infection and Immunity, University College London, Gower Street, London WC1E 6BT, UK Division of Advanced Therapies, National Institute for Biological Standards and Control, Blanche Lane, Potters Bar, Herts EN6 3QG, UK mary.collins@ucl.ac.uk.
authors.forename: Mary
authors.initials: M
authors.lastname: Collins
authors.affiliation: Institute of Child Health, University College London, Gower Street, London WC1E 6BT, UK.
authors.forename: Adrian
authors.initials: A
authors.lastname: Thrasher
mesh_terms: D016026:Bone Marrow Transplantation; D003141:Communicable Diseases / Q000235:genetics / Q000628:therapy; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D005822:Genetic Vectors; D002836:Hemophilia B / Q000235:genetics / Q000628:therapy; D006801:Humans; D009369:Neoplasms / Q000235:genetics / Q000628:therapy; D012162:Retinal Degeneration / Q000235:genetics / Q000628:therapy; D014780:Viruses / Q000235:genetics
keywords: evolutionary medicine; gene therapy; personalized medicine