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Gene Therapy for β-Hemoglobinopathies

Published:2017-05 Issue:5 Volume:25 Page:1142-1154
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Cavazzana Marina,Antoniani Chiara,Miccio Annarita

Funder

AFM-Telethon

European Research Council

Agence nationale de la recherche

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference88 articles.

1. The switch from fetal to adult hemoglobin;Sankaran;Cold Spring Harb. Perspect. Med.,2013

2. Haploinsufficiency for the erythroid transcription factor KLF1 causes hereditary persistence of fetal hemoglobin;Borg;Nat. Genet.,2010

3. HBS1L-MYB intergenic variants modulate fetal hemoglobin via long-range MYB enhancers;Stadhouders;J. Clin. Invest.,2014

4. The role of p22 NF-E4 in human globin gene switching;Zhou;J. Biol. Chem.,2004

5. The TR2 and TR4 orphan nuclear receptors repress Gata1 transcription;Tanabe;Genes Dev.,2007

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