Sickle Cell Disease in 2023: Laying the Foundation for Future Breakthroughs
Author:
Affiliation:
1. Division of Hematology, Department of Medicine, University of North Carolina School of Medicine, Chapel Hill, NC
Publisher
American Society of Hematology
Reference13 articles.
1. Global, regional, and national prevalence and mortality burden of sickle cell disease, 2000-–021: A systematic analysis from the Global Burden of Disease Study 2021;Thomson;Lancet Haematol,2023
2. Comparison of US federal and foundation funding of research for sickle cell disease and cystic fibrosis and factors associated with research productivity;Farooq;JAMA Network Open,2020
3. CRISPR-Cas9 editing of the HBG1 and HBG2 promoters to treat sickle cell disease;Sharma;N Engl J Med,2023
4. Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice;Everette;Nat Biomed Eng,2023
5. In vivo HSC prime editing rescues sickle cell disease in a mouse model;Georgakopoulou;Blood,2023
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