CRISPR–Cas9-mediated gene editing of the BCL11A enhancer for pediatric β0/β0 transfusion-dependent β-thalassemia
出版年份 2022 全文链接
标题
CRISPR–Cas9-mediated gene editing of the BCL11A enhancer for pediatric β0/β0 transfusion-dependent β-thalassemia
作者
关键词
-
出版物
NATURE MEDICINE
Volume -, Issue -, Pages -
出版商
Springer Science and Business Media LLC
发表日期
2022-08-04
DOI
10.1038/s41591-022-01906-z
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。- Reactivation of γ-globin expression through Cas9 or base editor to treat β-hemoglobinopathies
- (2020) Liren Wang et al. CELL RESEARCH
- Integrated single cell analysis of blood and cerebrospinal fluid leukocytes in multiple sclerosis
- (2020) David Schafflick et al. Nature Communications
- Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy
- (2020) Christian Brendel et al. Molecular Therapy-Methods & Clinical Development
- Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease
- (2020) Erica B. Esrick et al. NEW ENGLAND JOURNAL OF MEDICINE
- CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
- (2020) Haydar Frangoul et al. NEW ENGLAND JOURNAL OF MEDICINE
- Highly efficient therapeutic gene editing of human hematopoietic stem cells
- (2019) Yuxuan Wu et al. NATURE MEDICINE
- CRISPResso2 provides accurate and rapid genome editing sequence analysis
- (2019) Kendell Clement et al. NATURE BIOTECHNOLOGY
- Thalassaemia
- (2018) Ali T Taher et al. LANCET
- Dynamics of genetically engineered hematopoietic stem and progenitor cells after autologous transplantation in humans
- (2018) Serena Scala et al. NATURE MEDICINE
- In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases
- (2016) Luca Biasco et al. Cell Stem Cell
- Unperturbed vs. post-transplantation hematopoiesis
- (2016) Katrin Busch et al. CURRENT OPINION IN HEMATOLOGY
- The Present and Future Global Burden of the Inherited Disorders of Hemoglobin
- (2016) Frédéric B. Piel HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA
- Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype
- (2016) Christian Brendel et al. JOURNAL OF CLINICAL INVESTIGATION
- Hemoglobin switching's surprise: the versatile transcription factor BCL11A is a master repressor of fetal hemoglobin
- (2015) Daniel E Bauer et al. CURRENT OPINION IN GENETICS & DEVELOPMENT
- BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis
- (2015) Matthew C. Canver et al. NATURE
- Easy quantitative assessment of genome editing by sequence trace decomposition
- (2014) Eva K. Brinkman et al. NUCLEIC ACIDS RESEARCH
- An Erythroid Enhancer of BCL11A Subject to Genetic Variation Determines Fetal Hemoglobin Level
- (2013) D. E. Bauer et al. SCIENCE
- The Switch from Fetal to Adult Hemoglobin
- (2013) V. G. Sankaran et al. Cold Spring Harbor Perspectives in Medicine
- The Molecular Basis of -Thalassemia
- (2013) S. L. Thein Cold Spring Harbor Perspectives in Medicine
- Bcl11a is essential for lymphoid development and negatively regulates p53
- (2012) Yong Yu et al. JOURNAL OF EXPERIMENTAL MEDICINE
- Treatment and complications of thalassemia major in Guangxi, Southern China
- (2011) Xiao-Lin Yin et al. PEDIATRIC BLOOD & CANCER
- Beta-thalassemia
- (2010) Antonio Cao et al. GENETICS IN MEDICINE
- Specific Factors Influence the Success of Autologous and Allogeneic Hematopoietic Stem Cell Transplantation
- (2010) Thissiane L. Gonçalves et al. Oxidative Medicine and Cellular Longevity
- Genome-wide association study shows BCL11A associated with persistent fetal hemoglobin and amelioration of the phenotype of -thalassemia
- (2008) M. Uda et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
Discover Peeref hubs
Discuss science. Find collaborators. Network.
Join a conversationFind the ideal target journal for your manuscript
Explore over 38,000 international journals covering a vast array of academic fields.
Search