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

作者：Abou El Enein Mohamed^*; Bauer Gerhard; Reinke Petra; Renner Matthias; Schneider Christian K

来源：Trends in Molecular Medicine, 2014, 20(11): 632-642.

DOI：10.1016/j.molmed.2014.08.004

• gene therapy
• lentiviral vector: HIV
• Food and Drug Administration (FDA)
• European Medicines Agency (EMA)
• translational challenges

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.

• 出版日期2014-11