2014

A roadmap toward clinical translation of genetically-modified stem cells for treatment of HIV

Abou-El-Enein M, Bauer G, Reinke P, Renner M, Schneider C

Published in

Trends in molecular medicine: Volume 20, Issue 11, Page 632-42

Abstract

During the past decade, successful gene therapies for immunodeficiencies were finally brought to the clinic. This was accomplished through new gene therapy vectors and improved procedures for genetic modification of autologous hematopoietic stem cells. For HIV, autologous hematopoietic stem cell (HSC) gene therapy with 'anti-HIV genes' promises a functional cure for the disease. However, to develop such a therapy and translate it into a clinical application is rather challenging. The risks and benefits of such a therapy have to be understood, and regulatory hurdles need to be overcome. In this joint paper by academic researchers and regulators, we are, therefore, outlining a high level roadmap for the early stage development of HSC gene therapy as a potential functional cure for HIV.

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Cite this publication

DOI: 10.1016/j.molmed.2014.08.004