Detalhe da pesquisa
1.
Evaluation of both exonic and intronic variants for effects on RNA splicing allows for accurate assessment of the effectiveness of precision therapies.
PLoS Genet
; 16(10): e1009100, 2020 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-33085659
2.
Protospacer modification improves base editing of a canonical splice site variant and recovery of CFTR function in human airway epithelial cells.
Mol Ther Nucleic Acids
; 33: 335-350, 2023 Sep 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-37547293