Study of lentiviral vector for in utero gene transfer and functional analysis of human T-lymphotropic virus type p13(II)
Year: 2005
Type: article
Abstract: Human immunodeficiency virus-1 (HIV-1) based vectors are a promising approach for gene therapy targeting fetal tissues, however little is known about the role of accessory proteins in HIV-1 based gene transfer vectors in vivo.The impact of HIV-1 accessory proteins on gene transfer efficiency was analyzed in a murine in utero gene transfer model.Semi-quantitative PCR analysis revealed comparable levels of enhanced green fluorescent (EGFP) gene transfer by vectors, prepared in the presence or absence of HIV-1 accessory proteins, Nef, Vif, Vpu, and Vpr, in multiple organs systems, and immunoselected peripheral blood mononuclear cells (PBMCs).The EGFP transgene was detected in both mature myeloid and lymphoid cell lineages, as well as lineage marker negative marrow cells, with both vector preparations at all time points.Analysis of ex vivo expanded bone marrow-derived hematopoietic progenitor cells revealed successful gene transfer in both erythroid and myeloid lineages in the both vector groups, analyzed as early as one month of age.However, two months after gene transfer, the percentage of transduced hematopoietic progenitor colonies was significantly higher in animals that received accessory protein containing vectors.Animals that were administered vectors without accessory proteins had markedly reduced myeloid colonies transduced, resulting in a pronounced decrease in total levels of gene marking in blood
Author: Hajime Hiraragi
Institution: The Ohio State University
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: green
OpenAlex ID: W131739455