Format

Send to

Choose Destination
See comment in PubMed Commons below
Ann N Y Acad Sci. 2010 Aug;1202:134-40. doi: 10.1111/j.1749-6632.2010.05594.x.

A preclinical approach for gene therapy of beta-thalassemia.

Author information

1
Department of Pediatrics, Division of Hematology-Oncology, Weill Cornell Medical College, Children's Blood and Cancer Foundation Laboratories, New York, New York, USA.

Abstract

Lentiviral-mediated beta-globin gene transfer successfully treated beta-thalassemic mice. Based on this result, clinical trials were initiated. To date, however, no study has investigated the efficacy of gene therapy in relation to the nature of the different beta-globin mutations found in patients. Most mutations can be classified as beta(0) or beta(+), based on the amount of beta-globin protein produced. Therefore, we propose that a screening in vitro is necessary to verify the efficacy of gene transfer prior to treatment of individual patients. We used a two-phase liquid culture system to expand and differentiate erythroid progenitor cells (ErPCs) transduced with lentiviral vectors. We propose the use of this system to test the efficiency of lentiviral vectors carrying the human beta-globin gene, to correct the phenotype of ErPCs from patients preparing for gene therapy. This new approach might have profound implications for designing gene therapy and for understanding the genotype/phenotype variability observed in Cooley's anemia patients.

PMID:
20712784
PMCID:
PMC3068625
DOI:
10.1111/j.1749-6632.2010.05594.x
[Indexed for MEDLINE]
Free PMC Article
PubMed Commons home

PubMed Commons

0 comments
How to join PubMed Commons

    Supplemental Content

    Full text links

    Icon for Wiley Icon for PubMed Central
    Loading ...
    Support Center