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

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 su...

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Bibliographic Details
Main Authors: Grant D Trobridge, Brian C Beard, Robert A Wu, Christina Ironside, Punam Malik, Hans-Peter Kiem
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2012-01-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC3441638?pdf=render