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HIV-based lentiviral vectors: Origin and sequence differences

Nathan M. Johnson, Anna Francesca Alvarado, Trey N. Moffatt, Joshua M. Edavettal, Tarun A. Swaminathan, Stephen E. Braun

2021Molecular Therapy — Methods & Clinical Development39 citationsDOIOpen Access PDF

Abstract

regulatory elements were retained. With the use of representative vectors developed for clinical/commercial applications, we compared the vector backbone sequences to the initial sources of the HIV-1. All vectors included required elements: 5' long terminal repeat (LTR) through the Ψ packaging signal, central polypurine tract/chain termination sequence (cPPT/CTS), Rev responsive element (RRE), and 3' LTR, including a poly(A) signal. The Ψ signaling sequence demonstrated the greatest similarity between all vectors with only minor changes. The 3' LTR was the most divergent sequence with a range of deletions. The RRE length varied between vectors. Phylogenetic analysis of the cPPT/CTS indicated multiple sources, perhaps because of its later inclusion into lentiviral vector systems, whereas other regions revealed node clusters around the HIV-1 reference genomes HXB2 and NL4-3. We examine the function of each region in a lentiviral vector, the molecular differences between vectors, and where optimization may guide development of the lentiviral delivery systems.

Topics & Concepts

Computational biologyViral vectorBiologyLentivirusGenetic enhancementVector (molecular biology)GeneCoding regionLong terminal repeatGeneticsVirologyVirusGenomeRecombinant DNAViral diseaseVirus-based gene therapy researchCRISPR and Genetic EngineeringRNA Interference and Gene Delivery