CRISPR–Cas9-mediated gene editing of the BCL11A enhancer for pediatric β0/β0 transfusion-dependent β-thalassemia

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