From Mendel to a Mendelian disorder: towards a cure for sickle cell disease

Of mice and men: From hematopoiesis in mouse models to curative gene therapy for sickle cell disease

(2022) Julie Makani et al.   CELL

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

Gene Therapy in a Patient with Sickle Cell Disease

(2017) Jean-Antoine Ribeil et al.   NEW ENGLAND JOURNAL OF MEDICINE

Genetic variants at HbF-modifier loci moderate anemia and leukocytosis in sickle cell disease in Tanzania

(2014) Siana Nkya Mtatiro et al.   AMERICAN JOURNAL OF HEMATOLOGY

Enabling the genomic revolution in Africa

(2014) et al.   SCIENCE

A Functional Element Necessary for Fetal Hemoglobin Silencing

(2011) Vijay G. Sankaran et al.   NEW ENGLAND JOURNAL OF MEDICINE