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

Easy quantitative assessment of genome editing by sequence trace decomposition

Eva K. Brinkman, Tao Chen, Mario Amendola et al. · Nucleic Acids Research · 2014 · 2.6K citations · Full text

Life Sciences, Genetics, Molecular Biology +13

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

Haydar Frangoul, David Altshuler, Maria Domenica Cappellini et al. · New England Journal of Medicine · 2020 · 1.9K citations · Full text

Life Sciences, Genetics, Hematology +17

BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

Matthew C. Canver, Elenoe C. Smith, Falak Sher et al. · Nature · 2015 · 910 citations

Life Sciences, Genetics, Molecular Genetics +11

Genome-wide association study shows <i>BCL11A</i> associated with persistent fetal hemoglobin and amelioration of the phenotype of β-thalassemia

Manuela Uda, Renzo Galanello, Serena Sanna et al. · Proceedings of the National Academy of Sciences · 2008 · 672 citations · Full text

Life Sciences, Genetics, Molecular Genetics +17

An Erythroid Enhancer of <i>BCL11A</i> Subject to Genetic Variation Determines Fetal Hemoglobin Level

Daniel E. Bauer, Sophia C. Kamran, Samuel Lessard et al. · Science · 2013 · 667 citations · Full text

Life Sciences, Genetics, Molecular Genetics +17