Concepedia

Exagamglogene Autotemcel for Severe Sickle Cell Disease

Haydar Frangoul, Franco Locatelli, Akshay Sharma, Monica Bhatia, Markus Y. Mapara, Lyndsay Molinari, Donna A. Wall, Robert I. Liem, Paul Telfer, Ami J. Shah,

New England Journal of Medicine · 2024 · 304 citations · 13 references

DOI

Abstract

Treatment with exa-cel eliminated vaso-occlusive crises in 97% of patients with sickle cell disease for a period of 12 months or more. (CLIMB SCD-121; ClinicalTrials.gov number, NCT03745287.).

References

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 · 1.7K citations · Full text

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 · 645 citations · Full text

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A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease

Elliott Vichinsky, Carolyn Hoppe, Kenneth I. Ataga et al. · New England Journal of Medicine · 2019 · 581 citations · Full text

Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation

Éliane Gluckman, Barbara Cappelli, Françoise Bernaudin et al. · Blood · 2016 · 454 citations · Full text

The Switch from Fetal to Adult Hemoglobin

Vijay G. Sankaran, SH Orkin · Cold Spring Harbor Perspectives in Medicine · 2012 · 318 citations · Full text