Stem cell selection in vivo using foamy vectors cures canine pyruvate kinase deficiency.

ABSTRACT:

Background

Hematopoietic stem cell (HSC) gene therapy has cured immunodeficiencies including X-linked severe combined immunodeficiency (SCID-X1) and adenine deaminase deficiency (ADA). For these immunodeficiencies corrected cells have a selective advantage in vivo, and low numbers of gene-modified cells are sufficient to provide therapeutic benefit. Strategies to efficiently transduce and/or expand long-term repopulating cells in vivo are needed for treatment of diseases that require higher levels of corrected cells, such as hemoglobinopathies. Here we expanded corrected stem cells in vivo in a canine model of a severe erythroid disease, pyruvate kinase deficiency.

Methodology/principal findings

We used a foamy virus (FV) vector expressing the P140K mutant of methylguanine m

SUBMITTER: Trobridge GD 

PROVIDER: S-EPMC3441638 | biostudies-literature | 2012

REPOSITORIES: biostudies-literature