Detalhe da pesquisa
1.
Sickle Cell Disease: From Genetics to Curative Approaches.
Annu Rev Genomics Hum Genet
; 24: 255-275, 2023 08 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-37624668
2.
Adenine base editor-mediated correction of the common and severe IVS1-110 (G>A) ß-thalassemia mutation.
Blood
; 141(10): 1169-1179, 2023 03 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-36508706
3.
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression.
Nat Commun
; 13(1): 6618, 2022 11 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-36333351
4.
Severe axial and pelvifemoral muscle damage in immune-mediated necrotizing myopathy evaluated by whole-body MRI.
Semin Arthritis Rheum
; 50(6): 1437-1440, 2020 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-32222382
5.
Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype.
Sci Adv
; 6(7)2020 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-32917636