Detalhe da pesquisa
1.
Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy.
Mol Ther
; 29(11): 3243-3257, 2021 11 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-34509668
2.
Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy.
Hum Genet
; 135(9): 1029-40, 2016 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-27542949
3.
Transgenic overexpression of ribonucleotide reductase improves cardiac performance.
Proc Natl Acad Sci U S A
; 110(15): 6187-92, 2013 Apr 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-23530224
4.
Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy.
Nat Med
; 25(3): 427-432, 2019 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-30778238
5.
Genome engineering: a new approach to gene therapy for neuromuscular disorders.
Nat Rev Neurol
; 13(11): 647-661, 2017 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-28960187
6.
Generation and comparison of CRISPR-Cas9 and Cre-mediated genetically engineered mouse models of sarcoma.
Nat Commun
; 8: 15999, 2017 07 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-28691711