Hematopoietic-stem-cell-based gene therapy for HIV disease.
Although combination antiretroviral therapy can dramatically reduce the circulating viral load in those infected with HIV, replication-competent virus persists. To eliminate the need for indefinite treatment, there is growing interest in creating a functional HIV-resistant immune system through the use of gene-modified hematopoietic stem cells (HSCs). Proof of concept for this approach has been provided in the instance of an HIV-infected adult transplanted with allogeneic stem cells from a donor lacking the HIV coreceptor, CCR5. Here, we review this and other strategies for HSC-based gene therapy for HIV disease.
Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.
Hans-Peter
HP
Kiem
Keith R
KR
Jerome
Steven G
SG
Deeks
Joseph M
JM
McCune
D000328:Adult; D000818:Animals; D055786:Gene Knockout Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006678:HIV / Q000235:genetics / Q000502:physiology*; D015658:HIV Infections / Q000235:genetics / Q000276:immunology / Q000473:pathology / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007107:Immune System; D019713:Receptors, CCR5 / Q000235:genetics
D019713:Receptors, CCR5