Gene therapy: progress and predictions

被引：100

作者：

Collins, Mary ^{[1
,3
]}

Thrasher, Adrian ^{[2
]}

机构：

[1] UCL, Div Infect & Immun, London WC1E 6BT, England

[2] UCL, Inst Child Hlth, London WC1E 6BT, England

[3] Natl Inst Biol Stand & Controls, Div Adv Therapies, Potters Bar EN6 3QG, Herts, England

来源：

PROCEEDINGS OF THE ROYAL SOCIETY B-BIOLOGICAL SCIENCES | 2015年 / 282卷 / 1821期

关键词：

gene therapy; evolutionary medicine; personalized medicine; ADENOASSOCIATED VIRUS VECTORS; LEBERS CONGENITAL AMAUROSIS; HEMATOPOIETIC STEM-CELLS; ADENO-ASSOCIATED VIRUS; BETA-GLOBIN GENE; LENTIVIRAL VECTOR; EFFICIENT TRANSDUCTION; PARKINSONS-DISEASE; RETROVIRAL VECTORS; MAMMALIAN-CELLS;

D O I：

10.1098/rspb.2014.3003

中图分类号：

Q [生物科学];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

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.

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