Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia
出版年份 2017 全文链接
标题
Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia
作者
关键词
-
出版物
Nature Communications
Volume 8, Issue 1, Pages -
出版商
Springer Nature
发表日期
2017-08-30
DOI
10.1038/s41467-017-00479-7
参考文献
相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。- Emerging cellular and gene therapies for congenital anemias
- (2016) Leif S. Ludwig et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART C-SEMINARS IN MEDICAL GENETICS
- Customizing the genome as therapy for the β-hemoglobinopathies
- (2016) Matthew C. Canver et al. BLOOD
- Selective silencing of α-globin by the histone demethylase inhibitor IOX1: a potentially new pathway for treatment of β-thalassemia
- (2016) Sachith Mettananda et al. HAEMATOLOGICA
- Transcription factors LRF and BCL11A independently repress expression of fetal hemoglobin
- (2016) T. Masuda et al. SCIENCE
- Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells
- (2016) M. A. DeWitt et al. Science Translational Medicine
- Selective silencing of α-globin by the histone demethylase inhibitor IOX1: a potentially new pathway for treatment of β-thalassemia
- (2016) Sachith Mettananda et al. HAEMATOLOGICA
- Understanding α-globin gene regulation and implications for the treatment of β-thalassemia
- (2015) Sachith Mettananda et al. Annals of the New York Academy of Sciences
- -Globin as a molecular target in the treatment of -thalassemia
- (2015) S. Mettananda et al. BLOOD
- BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis
- (2015) Matthew C. Canver et al. NATURE
- Anemia: progress in molecular mechanisms and therapies
- (2015) Vijay G Sankaran et al. NATURE MEDICINE
- Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel
- (2014) E. Angelucci et al. HAEMATOLOGICA
- Targeted genome editing in human repopulating haematopoietic stem cells
- (2014) Pietro Genovese et al. NATURE
- Analysis of hundreds of cis-regulatory landscapes at high resolution in a single, high-throughput experiment
- (2014) Jim R Hughes et al. NATURE GENETICS
- Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV
- (2014) Pablo Tebas et al. NEW ENGLAND JOURNAL OF MEDICINE
- Lysine-specific demethylase 1 is a therapeutic target for fetal hemoglobin induction
- (2013) Lihong Shi et al. NATURE MEDICINE
- Genome engineering using the CRISPR-Cas9 system
- (2013) F Ann Ran et al. Nature Protocols
- A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
- (2012) M. Jinek et al. SCIENCE
- Thalassaemia
- (2011) Douglas R Higgs et al. LANCET
- Novel large deletions in the human α-globin gene cluster: Clarifying the HS-40 long-range regulatory role in the native chromosome environment
- (2010) Andreia Coelho et al. BLOOD CELLS MOLECULES AND DISEASES
- α-thalassaemia
- (2010) Cornelis L Harteveld et al. Orphanet Journal of Rare Diseases
- Chemical genetic strategy identifies histone deacetylase 1 (HDAC1) and HDAC2 as therapeutic targets in sickle cell disease
- (2010) James E. Bradner et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Association of globin gene quadruplication and heterozygous thalassemia in patients with thalassemia intermedia
- (2009) M. C. Sollaino et al. HAEMATOLOGICA
- Long-range regulation of ?? globin gene expression during erythropoiesis
- (2008) Douglas R Higgs et al. CURRENT OPINION IN HEMATOLOGY
- A large deletion in the human -globin cluster caused by a replication error is associated with an unexpectedly mild phenotype
- (2008) M. J. Rugless et al. HUMAN MOLECULAR GENETICS
Add your recorded webinar
Do you already have a recorded webinar? Grow your audience and get more views by easily listing your recording on Peeref.
Upload NowCreate your own webinar
Interested in hosting your own webinar? Check the schedule and propose your idea to the Peeref Content Team.
Create Now