Related references
Note: Only part of the references are listed.CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
H. Frangoul et al.
NEW ENGLAND JOURNAL OF MEDICINE (2021)
Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype
Leslie Weber et al.
SCIENCE ADVANCES (2020)
A natural regulatory mutation in the proximal promoter elevates fetal globin expression by creating a de novo GATA1 site
Gabriella E. Martyn et al.
BLOOD (2019)
Genome editing of HBG1 and HBG2 to induce fetal hemoglobin
Jean-Yves Metais et al.
BLOOD ADVANCES (2019)
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
A. A. Thompson et al.
NEW ENGLAND JOURNAL OF MEDICINE (2018)
Wake-up Sleepy Gene: Reactivating Fetal Globin for beta-Hemoglobinopathies
Beeke Wienert et al.
TRENDS IN GENETICS (2018)
Gene Therapy in a Patient with Sickle Cell Disease
Jean-Antoine Ribeil et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
KLF1 drives the expression of fetal hemoglobin in British HPFH
Beeke Wienert et al.
BLOOD (2017)
A Genetic Variant Ameliorates β-Thalassemia Severity by Epigenetic-Mediated Elevation of Human Fetal Hemoglobin Expression
Diyu Chen et al.
AMERICAN JOURNAL OF HUMAN GENETICS (2017)
New trend in the epidemiology of thalassaemia
Chi-Kong Li
BEST PRACTICE & RESEARCH CLINICAL OBSTETRICS & GYNAECOLOGY (2017)
A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition
Elizabeth A. Traxler et al.
NATURE MEDICINE (2016)
Editing the genome to introduce a beneficial naturally occurring mutation associated with increased fetal globin
Beeke Wienert et al.
NATURE COMMUNICATIONS (2015)
Isolation of Single Human Hematopoietic Stem Cells Capable of Long-Term Multilineage Engraftment
Faiyaz Notta et al.
SCIENCE (2011)
Share Paper
