HIV-1 vectors: fulfillment of expectations, further advancements, and still a way to go.
The ability of lentiviral vectors to transduce and stably integrate their genomes into non-dividing cells was the major reason for the development of the HIV-1 based vector gene delivery system. The first VSV-G pseudotyped lentiviral vectors fulfilled these expectations by ferrying large genetic payloads to non-dividing cells in vitro and in vivo. Here we discuss advances in HIV-1 vector systems which lead to improvement in biosafety, transduction efficiency, longevity and regulation of transgene expression, and vector production. The successful use of the advanced HIV-1 based vector system opened new avenues in establishing transgenic animal models for basic research. Additionally, we describe accomplishments using HIV-1 based vectors to correct pathological courses of incurable diseases in preclinical animal models including Parkinson's disease and beta-thalassemia.
Cockrell
Adam S
AS
Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Kafri
Tal
T
D000818:Animals; D030801:Animals, Genetically Modified; D004195:Disease Models, Animal; D015870:Gene Expression; D005786:Gene Expression Regulation; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D015497:HIV-1 / Q000235:genetics*; D009154:Mutation; D014161:Transduction, Genetic; D016662:Virus Integration / Q000235:genetics; D014779:Virus Replication / Q000235:genetics