Detalhe da pesquisa
1.
Base editing of key residues in the BCL11A-XL-specific zinc finger domains derepresses fetal globin expression.
Mol Ther
; 32(3): 663-677, 2024 Mar 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-38273654
2.
Precise correction of a spectrum of ß-thalassemia mutations in coding and non-coding regions by base editors.
Mol Ther Nucleic Acids
; 35(2): 102205, 2024 Jun 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-38817682
3.
Genome Engineering of Hematopoietic Stem Cells Using CRISPR/Cas9 System.
Methods Mol Biol
; 2429: 307-331, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-35507170
4.
Erythroid lineage-specific lentiviral RNAi vectors suitable for molecular functional studies and therapeutic applications.
Sci Rep
; 12(1): 14033, 2022 08 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-35982069
5.
Efficient and error-free correction of sickle mutation in human erythroid cells using prime editor-2.
Front Genome Ed
; 4: 1085111, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-36605051
6.
Identification of novel HPFH-like mutations by CRISPR base editing that elevate the expression of fetal hemoglobin.
Elife
; 112022 02 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-35147495