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

Treating Rare Diseases in Africa: The Drugs Exist but the Need Is Unmet

(2022) Lucio Luzzatto et al.   Frontiers in Pharmacology

Transcription factor competition at the γ-globin promoters controls hemoglobin switching

(2021) Nan Liu et al.   NATURE GENETICS

A unified model of human hemoglobin switching through single-cell genome editing

(2021) Yong Shen et al.   Nature Communications

Reactivation of a developmentally silenced embryonic globin gene

(2021) Andrew J. King et al.   Nature Communications

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