2010/02/08 by David Escors, Karine Breckpot · 1 citation
Biochemistry, Genetics and Molecular Biology · #CRISPR and Genetic Engineering #RNA Interference and Gene Delivery #Virus-based gene therapy research
paper · doi:10.1007/s00005-010-0063-4
openalex publication_date 2010/02/08 · crossref created 2010/02/08 · crossref issued 2010/02/09 · crossref published 2010/02/09 · crossref published-online 2010/02/09 · crossref published-print 2010/04/01 · crossref deposited 2024/01/02 · openalex created_date 2025/10/10 · crossref indexed 2026/07/30 · openalex updated_date 2026/07/31
The concept of gene therapy originated in the mid twentieth century and was perceived as a revolutionary technology with the promise to cure almost any disease of which the molecular basis was understood. Since then, several gene vectors have been developed and the feasibility of gene therapy has been shown in many animal models of human disease. However, clinical efficacy could not be demonstrated until the beginning of the new century in a small-scale clinical trial curing an otherwise fatal immunodeficiency disorder in children. This first success, achieved after retroviral therapy, was later overshadowed by the occurrence of vector-related leukemia in a significant number of the treated children, demonstrating that the future success of gene therapy depends on our understanding of vector biology. This has led to the development of later-generation vectors with improved efficiency, specificity, and safety. Amongst these are HIV-1 lentivirus-based vectors (lentivectors), which are being increasingly used in basic and applied research. Human gene therapy clinical trials are currently underway using lentivectors in a wide range of human diseases. The intention of this review is to describe the main scientific steps leading to the engineering of HIV-1 lentiviral vectors and place them in the context of current human gene therapy.