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CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
N Engl J Med. 2021 Jan 21;384(3):252-260. doi: 10.1056/NEJMoa2031054. Epub 2020 Dec 5.
N Engl J Med. 2021.
PMID: 33283989
Clinical Trial.
Rapid progression and mortality of lysosomal acid lipase deficiency presenting in infants.
Jones SA, Valayannopoulos V, Schneider E, Eckert S, Banikazemi M, Bialer M, Cederbaum S, Chan A, Dhawan A, Di Rocco M, Domm J, Enns GM, Finegold D, Gargus JJ, Guardamagna O, Hendriksz C, Mahmoud IG, Raiman J, Selim LA, Whitley CB, Zaki O, Quinn AG.
Jones SA, et al.
Genet Med. 2016 May;18(5):452-8. doi: 10.1038/gim.2015.108. Epub 2015 Aug 27.
Genet Med. 2016.
PMID: 26312827
Free PMC article.
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