title: A roadmap toward clinical translation of genetically-modified stem cells for treatment of HIV.
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.
authors.affiliation: Berlin-Brandenburg Center for Regenerative Therapies (BCRT), Charité University Medicine Berlin, Campus Virchow, Berlin, Germany; Department of Nephrology and Internal Intensive Care, Charité University Medicine Berlin, Campus Virchow, Berlin, Germany. Electronic address: Mohamed.abou-el-enein@charite.de.
authors.forename: Mohamed
authors.initials: M
authors.lastname: Abou-El-Enein
authors.affiliation: University of California Davis, Institute For Regenerative Cures (IRC) Sacramento, CA, USA.
authors.forename: Gerhard
authors.initials: G
authors.lastname: Bauer
authors.affiliation: Berlin-Brandenburg Center for Regenerative Therapies (BCRT), Charité University Medicine Berlin, Campus Virchow, Berlin, Germany; Department of Nephrology and Internal Intensive Care, Charité University Medicine Berlin, Campus Virchow, Berlin, Germany.
authors.forename: Petra
authors.initials: P
authors.lastname: Reinke
authors.affiliation: Paul-Ehrlich-Institut, Paul-Ehrlich-Str. 51-59, D-63225 Langen, Germany.
authors.forename: Matthias
authors.initials: M
authors.lastname: Renner
authors.affiliation: Formerly Committee for Advanced Therapies, European Medicines Agency, 7, Westferry Circus E14 4HB, London, UK; Danish Health and Medicines Authority, Axel Heides Gade 1, 2300 Copenhagen, Denmark; Twincore Centre for Experimental and Clinical Infection Research, Feodor-Lynen-Straße 730625 Hannover, Germany.
authors.forename: Christian K
authors.initials: CK
authors.lastname: Schneider
mesh_terms: D000818:Animals; D064987:Cell- and Tissue-Based Therapy / Q000009:adverse effects / Q000379:methods / Q000592:standards; D004195:Disease Models, Animal; D015316:Genetic Therapy / Q000009:adverse effects / Q000379:methods / Q000592:standards; D015658:HIV Infections / Q000235:genetics* / Q000628:therapy*; D006801:Humans; D013234:Stem Cells / Q000378:metabolism*; D057170:Translational Research, Biomedical
keywords: European Medicines Agency (EMA); Food and Drug Administration (FDA); gene therapy; lentiviral vector: HIV; translational challenges