Transcription factors LRF and BCL11A independently repress expression of fetal hemoglobin-Reference-Cited by-同舟云学术

Transcription factors LRF and BCL11A independently repress expression of fetal hemoglobin

Published:2016-01-15 Issue:6270 Volume:351 Page:285-289
ISSN:0036-8075
Container-title:Science
language:en
Short-container-title:Science

Author:

Masuda Takeshi¹,Wang Xin²,Maeda Manami¹,Canver Matthew C.³,Sher Falak³,Funnell Alister P. W.⁴,Fisher Chris⁵,Suciu Maria⁵,Martyn Gabriella E.⁴,Norton Laura J.⁴,Zhu Catherine¹,Kurita Ryo⁶,Nakamura Yukio⁶⁷,Xu Jian³⁸,Higgs Douglas R.⁵,Crossley Merlin⁴,Bauer Daniel E.³,Orkin Stuart H.³⁹,Kharchenko Peter V.²,Maeda Takahiro¹

Affiliation:

1. Division of Hematology, Department of Medicine, Brigham and Women’s Hospital, Harvard Medical School, Boston, MA 02115, USA.

2. Department of Biomedical Informatics, Harvard Medical School, Boston, MA 02115, USA.

3. Division of Hematology/Oncology, Boston Children’s Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Stem Cell Institute, Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA.

4. School of Biotechnology and Biomolecular Sciences, University of New South Wales, Sydney, NSW 2052, Australia.

5. Medical Research Council, Molecular Haematology Unit, Weatherall Institute of Molecular Medicine, Oxford University, Oxford, UK.

6. Cell Engineering Division, RIKEN BioResource Center, Tsukuba, Ibaraki, Japan.

7. Comprehensive Human Sciences, University of Tsukuba, Tsukuba, Ibaraki, Japan.

8. Children’s Research Institute, Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.

9. Howard Hughes Medical Institute, Boston, MA 02115, USA.

Abstract

Reactivating the fetal globin gene Mutation of adult-type globin genes causes sickle cell disease and thalassemia. Although treating these hemoglobinopathies with gene therapy is possible, there is a pressing need for pharmacologic approaches to treat general patient populations. One promising approach is to reactivate repressed expression of fetal-type hemoglobin (HbF) in adult erythroid cells. Masuda et al. reveal a molecular mechanism governing HbF repression as mediated by the LRF/ZBTB7A transcription factor. The study may encourage the development of new HbF reactivation therapies for hemoglobinopathies. Science , this issue p. 285

Funder

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

NIDDK Career Development

Doris Duke Charitable Foundation Innovations in Clinical Research

Ellison Medical Foundation

NIH

American Society of Hematology Bridge Program

Publisher

American Association for the Advancement of Science (AAAS)

Subject

Multidisciplinary

Reference63 articles.