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

Mohamed Abou-El-Enein, Gerhard Bauer, Petra Reinke, Matthias Renner, Christian K. Schneider

Research output: Contribution to journalArticle

18 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)632-642
Number of pages11
JournalTrends in Molecular Medicine
Volume20
Issue number11
DOIs
StatePublished - Nov 1 2014

Keywords

  • European medicines agency (EMA)
  • Food and drug administration (FDA)
  • Gene therapy
  • Lentiviral vector: HIV
  • Translational challenges

ASJC Scopus subject areas

  • Molecular Biology
  • Molecular Medicine

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