Author： Blomberg Pontus Smith CI Edvard
Publisher： Informa Healthcare
ISSN： 1471-2598
Source： Expert Opinion on Biological Therapy, Vol.3, Iss.6, 2003-09, pp. : 941-949
Disclaimer: Any content in publications that violate the sovereignty, the constitution or regulations of the PRC is not accepted or approved by CNPIEC.
Abstract
The concept of gene therapy involves the introduction of genetic material into patient cells to cure or alleviate the symptoms of a disease by complementing a damaged gene or by giving the cell a new function. The belief that gene therapy would soon reach the clinic has been widely spread, frequently resulting in controversies when these expectations were not met. Nevertheless, over the last 10-year period, the experience from a number of clinical trials has taught us that gene transfer is technically feasible, but that the gene delivery vehicles, or vectors, for the transfer of genetic material are still suboptimal and that treatment may have severe side effects. This review will provide examples of different genetic disorders for which gene therapy is an option and has been attempted. It will also briefly discuss the existing vector systems and mention their advantages and drawbacks.
Related content
Access to resources
Recommend
Gene therapy for lipoprotein disorders
By Broedl Uli C Rader Daniel J
Expert Opinion on Biological Therapy, Vol. 5, Iss. 8, 2005-08 ,pp. :
Gene therapy for renal disorders
By Lien Yeong-Hau H Lai Li-Wen
Expert Opinion on Biological Therapy, Vol. 4, Iss. 6, 2004-06 ,pp. :
Gene therapy for lysosomal storage disorders
By Barranger John A Novelli Enrico M
Expert Opinion on Biological Therapy, Vol. 1, Iss. 5, 2001-09 ,pp. :
Gene therapy for lysosomal storage disorders
Expert Opinion on Biological Therapy, Vol. 11, Iss. 9, 2011-09 ,pp. :