Detalhe da pesquisa
1.
Functional restoration of a CFTR splicing mutation through RNA delivery of CRISPR adenine base editor.
Mol Ther
; 31(6): 1647-1660, 2023 06 07.
Artigo
Inglês
| MEDLINE | ID: mdl-36895161
2.
Transcriptomic analysis of CFTR-impaired endothelial cells reveals a pro-inflammatory phenotype.
Eur Respir J
; 57(4)2021 04.
Artigo
Inglês
| MEDLINE | ID: mdl-33184117
3.
Prime editing functionally corrects cystic fibrosis-causing CFTR mutations in human organoids and airway epithelial cells.
Cell Rep Med
; 5(5): 101544, 2024 May 21.
Artigo
Inglês
| MEDLINE | ID: mdl-38697102
4.
Nanoblades allow high-level genome editing in murine and human organoids.
Mol Ther Nucleic Acids
; 33: 57-74, 2023 Sep 12.
Artigo
Inglês
| MEDLINE | ID: mdl-37435135
5.
One Size Does Not Fit All: The Past, Present and Future of Cystic Fibrosis Causal Therapies.
Cells
; 11(12)2022 06 08.
Artigo
Inglês
| MEDLINE | ID: mdl-35740997
6.
Novel CFTR modulator combinations maximise rescue of G85E and N1303K in rectal organoids.
ERJ Open Res
; 8(2)2022 Apr.
Artigo
Inglês
| MEDLINE | ID: mdl-35449760
7.
Severity of the S1251N allele in cystic fibrosis is affected by the presence of the F508C variant in cis.
J Cyst Fibros
; 21(4): 644-651, 2022 07.
Artigo
Inglês
| MEDLINE | ID: mdl-35690578
8.
Rewriting CFTR to cure cystic fibrosis.
Prog Mol Biol Transl Sci
; 182: 185-224, 2021.
Artigo
Inglês
| MEDLINE | ID: mdl-34175042
9.
On the Corner of Models and Cure: Gene Editing in Cystic Fibrosis.
Front Pharmacol
; 12: 662110, 2021.
Artigo
Inglês
| MEDLINE | ID: mdl-33986686
10.
Phenotyping of Rare CFTR Mutations Reveals Distinct Trafficking and Functional Defects.
Cells
; 9(3)2020 03 19.
Artigo
Inglês
| MEDLINE | ID: mdl-32204475