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Updated: Aug 20, 2026

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
Published on: September 5, 2016
Harnessing HIV for therapy, basic research and biotechnology
Maciej Wiznerowicz1, Didier Trono
1Frontiers in Genetics, National Center for Competence in Research, School of Life Sciences, Swiss Federal Institute of Technology, Laussane, Switzerland.
Abstract:
First described about a decade ago, lentiviral vectors ('lentivectors') have emerged as potent and versatile tools of gene transfer for basic and applied research and offer exciting perspectives for the field of gene therapy. In the clinic, HIV-based vectors are showing particular promise for delivering therapeutic genes to hematopoietic stem cells (HSCs) and terminally differentiated targets in the central nervous system (CNS). Their flexible design facilitates the accommodation of sophisticated elements of control for the precise tuning of transgene expression. The delivery of small interfering RNAs (siRNAs) and genomic or cDNA libraries and the creation of transgenic animals are the most recent and exciting applications of HIV-based vectors that will help to tackle fundamental issues across wide areas of biology.
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