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.
journal_name
Trends Mol Medjournal_title
Trends in molecular medicineauthors
Abou-El-Enein M,Bauer G,Reinke P,Renner M,Schneider CKdoi
10.1016/j.molmed.2014.08.004subject
Has Abstractpub_date
2014-11-01 00:00:00pages
632-42issue
11eissn
1471-4914issn
1471-499Xpii
S1471-4914(14)00137-3journal_volume
20pub_type
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