Detalhe da pesquisa
1.
Extru-seq: a method for predicting genome-wide Cas9 off-target sites with advantages of both cell-based and in vitro approaches.
Genome Biol;
24(1): 4, 2023 Jan 10.
Artigo
em Inglês
| MEDLINE
| ID: mdl-36627653
2.
Genome editing-mediated knock-in of therapeutic genes ameliorates the disease phenotype in a model of hemophilia.
Mol Ther Nucleic Acids;
29: 551-562, 2022 Sep 13.
Artigo
em Inglês
| MEDLINE
| ID: mdl-36090746
3.
In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy.
Sci Adv;
8(3): eabj6901, 2022 01 21.
Artigo
em Inglês
| MEDLINE
| ID: mdl-35061543
4.
CRISPR-Cas9-mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis.
Sci Adv;
5(10): eaax1210, 2019 10.
Artigo
em Inglês
| MEDLINE
| ID: mdl-31692906