Abstract
Get full access to this article
View all access options for this article.
References
1.
Weatherall
DJ
. Phenotype-genotype relationships in monogenic disease: lessons from the thalassaemias . Nat Rev Genet , 2001 ; 2 :245 –255 .
2.
Frangoul
H
,
Altshuler
D
,
Cappellini
MD
, et al. CRISPR-Cas9 gene editing for sickle cell disease and beta-Thalassemia . N Engl J Med , 2021 ; 384 :252 –260 .
3.
Newby
GA
,
Yen
JS
,
Woodard
KJ
, et al. Base editing of haematopoietic stem cells rescues sickle cell disease in mice . Nature , 2021 ; 595 :295 –302 .