Detalhe da pesquisa
1.
Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus.
Mol Ther
; 30(1): 223-237, 2022 01 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-33794364
2.
AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9.
Mol Ther
; 28(6): 1432-1441, 2020 06 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-32348718
3.
Highly efficient editing of the ß-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease.
Nucleic Acids Res
; 47(15): 7955-7972, 2019 09 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-31147717
4.
CD7-edited T cells expressing a CD7-specific CAR for the therapy of T-cell malignancies.
Blood
; 130(3): 285-296, 2017 07 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-28539325
5.
Examination of CRISPR/Cas9 design tools and the effect of target site accessibility on Cas9 activity.
Exp Physiol
; 103(4): 456-460, 2018 04 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-28303677
6.
Streptococcus thermophilus CRISPR-Cas9 Systems Enable Specific Editing of the Human Genome.
Mol Ther
; 24(3): 636-44, 2016 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-26658966
7.
Comprehensive analysis and accurate quantification of unintended large gene modifications induced by CRISPR-Cas9 gene editing.
Sci Adv
; 8(42): eabo7676, 2022 Oct 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-36269834
8.
Tools for experimental and computational analyses of off-target editing by programmable nucleases.
Nat Protoc
; 16(1): 10-26, 2021 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-33288953
9.
Design and Validation of CRISPR/Cas9 Systems for Targeted Gene Modification in Induced Pluripotent Stem Cells.
Methods Mol Biol
; 1498: 3-21, 2017.
Artigo
em Inglês
| MEDLINE | ID: mdl-27709565