The use of cell-delivered gene therapy for the treatment of HIV/AIDS.
HIV/AIDS is a disease that impairs immune function, primarily by decreasing T-lymphocyte count. Its progression can be contained by highly active antiretroviral therapy (HAART), but there are side effects that can be severe, and the development of resistance often forces the physician to modify the HAART regimen. There are no vaccines available for HIV. An alternative approach that could provide a path to a curative therapy is the use of cell-delivered gene therapy in which an anti-HIV gene(s) is introduced into hematopoietic cells to produce a population that is protected from the effects of HIV. In this paper, we review the field and discuss an approach using a short hairpin RNA to CCR5, an important co-receptor for HIV.
Symonds
Geoff P
GP
UCLA Department of Microbiology, Immunology and Molecular Genetics, UCLA AIDS Institute, 10940 Wilshire Blvd., Los Angeles, CA 90024-1678, USA. geoff.symonds@calimmuneinc.com
Johnstone
Helen A
HA
Millington
Michelle L
ML
Boyd
Maureen P
MP
Burke
Bryan P
BP
Breton
Louis R
LR
D000818:Animals; D002986:Clinical Trials as Topic; D004195:Disease Models, Animal; D005814:Genes, Viral / Q000235:genetics; D015316:Genetic Therapy / Q000639:trends; D006678:HIV / Q000472:pathogenicity / Q000502:physiology*; D015658:HIV Infections / Q000235:genetics / Q000276:immunology / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation; D006412:Hematopoietic Stem Cells / Q000378:metabolism* / Q000473:pathology; D006801:Humans; D051379:Mice; D034741:RNA, Small Interfering / Q000235:genetics / Q000627:therapeutic use*; D019713:Receptors, CCR5 / Q000235:genetics / Q000378:metabolism*; D015728:Receptors, HIV / Q000235:genetics / Q000378:metabolism*; D014779:Virus Replication / Q000235:genetics
D034741:RNA, Small Interfering; D019713:Receptors, CCR5; D015728:Receptors, HIV