학술논문

Phase I/II Clinical Trials Using Gene-Modified Adult Hematopoietic Stem Cells for HIV: Lessons Learnt
Document Type
article
Source
Subject
Biochemistry and Cell Biology
Biological Sciences
Clinical Trials and Supportive Activities
Gene Therapy
Transplantation
Infectious Diseases
Stem Cell Research - Nonembryonic - Human
HIV/AIDS
Stem Cell Research
Genetics
Regenerative Medicine
Clinical Research
Development of treatments and therapeutic interventions
Evaluation of treatments and therapeutic interventions
5.2 Cellular and gene therapies
6.2 Cellular and gene therapies
Infection
Clinical Sciences
Biochemistry and cell biology
Language
Abstract
Gene therapy for individuals infected with HIV has the potential to provide a once-only treatment that will act to reduce viral load, preserve the immune system, and mitigate cumulative toxicities associated with highly active antiretroviral therapy (HAART). The authors have been involved in two clinical trials (phase I and phase II) using gene-modified adult hematopoietic stem cells (HSCs), and these are discussed as prototypic trials within the general field of HSC gene therapy trials for HIV. Taken as a group these trials have shown (i) the safety of both the procedure and the anti-HIV agents themselves and (ii) the feasibility of the approach. They point to the requirement for (i) the ability to transduce and infuse as many as possible gene-containing HSC and/or (ii) high engraftment and in vivo expansion of these cells, (iii) potentially increased efficacy of the anti-HIV agent(s) and (iv) automation of the cell processing procedure.