Transduction of long-term and mobilized peripheral blood-derived NOD/SCID repopulating cells by foamy virus vectors.

Abstract:

:Foamy virus (FV) vectors are a promising gene delivery system for use in hematopoietic stem cell gene therapy. Previous FV vector marking studies in the NOD/SCID xenotransplantation model used umbilical cord blood (UCB)-derived SCID repopulating cells (SRCs) that were assayed 5-10 weeks posttransplantation. We now report efficient FV vector transduction (>65%) of UCB-derived primitive, long-term SRCs engrafted for 18 weeks. In addition, we evaluated gene transfer into mobilized peripheral blood (MPB)-derived SRCs by improved, deleted FV vectors containing minimal cis-acting sequences and packaged by split helper constructs that would be appropriate for use in clinical trials. When used at a multiplicity of infection of 1 in a 10-hr transduction protocol, these improved vectors transduced 34% of engrafted MPB-derived SRCs.

journal_name

Hum Gene Ther

journal_title

Human gene therapy

authors

Josephson NC,Trobridge G,Russell DW

doi

10.1089/10430340460732481

keywords:

subject

Has Abstract

pub_date

2004-01-01 00:00:00

pages

87-92

issue

1

eissn

1043-0342

issn

1557-7422

journal_volume

15

pub_type

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