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

15 Citations (Scopus)

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

Fingerprint

Genetic Therapy
Stem Cells
Hematopoietic Stem Cells
HIV
Cell- and Tissue-Based Therapy
Therapeutics
Joints
Research Personnel
Genes

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

Cite this

A roadmap toward clinical translation of genetically-modified stem cells for treatment of HIV. / Abou-El-Enein, Mohamed; Bauer, Gerhard; Reinke, Petra; Renner, Matthias; Schneider, Christian K.

In: Trends in Molecular Medicine, Vol. 20, No. 11, 01.11.2014, p. 632-642.

Research output: Contribution to journalArticle

Abou-El-Enein, Mohamed ; Bauer, Gerhard ; Reinke, Petra ; Renner, Matthias ; Schneider, Christian K. / A roadmap toward clinical translation of genetically-modified stem cells for treatment of HIV. In: Trends in Molecular Medicine. 2014 ; Vol. 20, No. 11. pp. 632-642.
@article{51ec287f6d944c3caee374763e7f0e06,
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.",
keywords = "European medicines agency (EMA), Food and drug administration (FDA), Gene therapy, Lentiviral vector: HIV, Translational challenges",
author = "Mohamed Abou-El-Enein and Gerhard Bauer and Petra Reinke and Matthias Renner and Schneider, {Christian K.}",
year = "2014",
month = "11",
day = "1",
doi = "10.1016/j.molmed.2014.08.004",
language = "English (US)",
volume = "20",
pages = "632--642",
journal = "Trends in Molecular Medicine",
issn = "1471-4914",
publisher = "Elsevier Limited",
number = "11",

}

TY - JOUR

T1 - A roadmap toward clinical translation of genetically-modified stem cells for treatment of HIV

AU - Abou-El-Enein, Mohamed

AU - Bauer, Gerhard

AU - Reinke, Petra

AU - Renner, Matthias

AU - Schneider, Christian K.

PY - 2014/11/1

Y1 - 2014/11/1

N2 - 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.

AB - 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.

KW - European medicines agency (EMA)

KW - Food and drug administration (FDA)

KW - Gene therapy

KW - Lentiviral vector: HIV

KW - Translational challenges

UR - http://www.scopus.com/inward/record.url?scp=84909644152&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84909644152&partnerID=8YFLogxK

U2 - 10.1016/j.molmed.2014.08.004

DO - 10.1016/j.molmed.2014.08.004

M3 - Article

VL - 20

SP - 632

EP - 642

JO - Trends in Molecular Medicine

JF - Trends in Molecular Medicine

SN - 1471-4914

IS - 11

ER -