Detalhe da pesquisa
1.
Defective membrane fusion and repair in Anoctamin5-deficient muscular dystrophy.
Hum Mol Genet
; 25(10): 1900-1911, 2016 05 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-26911675
2.
Systemic AAV-Mediated ß-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice.
Mol Ther
; 25(4): 855-869, 2017 04 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-28284983
3.
Vascular delivery of rAAVrh74.MCK.GALGT2 to the gastrocnemius muscle of the rhesus macaque stimulates the expression of dystrophin and laminin α2 surrogates.
Mol Ther
; 22(4): 713-24, 2014 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-24145553
4.
AAV-mediated overexpression of human α7 integrin leads to histological and functional improvement in dystrophic mice.
Mol Ther
; 21(3): 520-5, 2013 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-23319059
5.
Expression and function of four AAV-based constructs for dystrophin restoration in the mdx mouse model of Duchenne muscular dystrophy.
Biol Open
; 12(9)2023 09 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-37670674
6.
Preclinical Systemic Delivery of Adeno-Associated α-Sarcoglycan Gene Transfer for Limb-Girdle Muscular Dystrophy.
Hum Gene Ther
; 32(7-8): 390-404, 2021 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-33349138
7.
Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the mdx Mouse Model of Duchenne Muscular Dystrophy.
Hum Gene Ther
; 32(7-8): 375-389, 2021 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-33397205
8.
AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD.
JCI Insight
; 3(22)2018 11 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-30429376
9.
Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy.
Hum Gene Ther
; 29(7): 749-762, 2018 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-28707952
10.
MicroRNA-29 overexpression by adeno-associated virus suppresses fibrosis and restores muscle function in combination with micro-dystrophin.
JCI Insight
; 2(9)2017 May 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-28469083
11.
Human α7 Integrin Gene (ITGA7) Delivered by Adeno-Associated Virus Extends Survival of Severely Affected Dystrophin/Utrophin-Deficient Mice.
Hum Gene Ther
; 26(10): 647-56, 2015 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-26076707
12.
AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models.
Ann Clin Transl Neurol
; 2(3): 256-70, 2015 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-25815352
13.
Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.
Nat Med
; 20(9): 992-1000, 2014 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-25108525
14.
Characterization of bone resorption in novel in vitro and in vivo models of oral squamous cell carcinoma.
Oral Oncol
; 48(6): 491-9, 2012 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-22265717
15.
Corrigendum: Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.
Nat Med
; 21(5): 537, 2015 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-25951531
16.
Corrigendum: Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.
Nat Med
; 21(4): 414, 2015 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-25849273