Abstract
Background HIV-1 is still a major public health problem and one of the priorities of the World Health Organization. The development of HAART against HIV was a considerable advance for infected individuals, but this life-long treatment does only block virus replication, and no viral eradication is obtained. Furthermore, HAART may exhibit long-term toxicity and may eventually lead to the emergence of drug-resistant viral variants. We explore a new durable therapeutic intervention based on a gene therapy that induces RNA interference (RNAi) against HIV1. In this pre-clinical research setting, “humanized” experimental mouse models are of interest considering the relative ease of handling and relatively low cost as compared to non-human primates. Methods
Highlights
HIV-1 is still a major public health problem and one of the priorities of the World Health Organization
We explore a new durable therapeutic intervention based on a gene therapy that induces RNA interference (RNAi) against HIV1
We have developed an RNAi gene therapy based on the transduction of human hematopoietic progenitor cells (HPC) with lentiviral vectors encoding short-hairpin RNAs to induce silencing of HIV genes
Summary
HIV-1 is still a major public health problem and one of the priorities of the World Health Organization. The development of HAART against HIV was a considerable advance for infected individuals, but this life-long treatment does only block virus replication, and no viral eradication is obtained. HAART may exhibit long-term toxicity and may eventually lead to the emergence of drug-resistant viral variants. We explore a new durable therapeutic intervention based on a gene therapy that induces RNA interference (RNAi) against HIV1. In this pre-clinical research setting, “humanized” experimental mouse models are of interest considering the relative ease of handling and relatively low cost as compared to non-human primates
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