Detalhe da pesquisa
1.
Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial.
Muscle Nerve
; 69(1): 93-98, 2024 Jan.
Artigo
Inglês
| MEDLINE | ID: mdl-37577753
2.
AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1.
Gene Ther
; 29(3-4): 127-137, 2022 04.
Artigo
Inglês
| MEDLINE | ID: mdl-33542455
3.
Vasculitic neuropathy associated with IgG4-related kidney disease: A case report and literature review.
Clin Nephrol
; 96(3): 175-179, 2021 Sep.
Artigo
Inglês
| MEDLINE | ID: mdl-34085635
4.
AAV1.NT-3 gene therapy increases muscle fiber diameter through activation of mTOR pathway and metabolic remodeling in a CMT mouse model.
Gene Ther
; 25(2): 129-138, 2018 Apr.
Artigo
Inglês
| MEDLINE | ID: mdl-29523879
5.
Defective membrane fusion and repair in Anoctamin5-deficient muscular dystrophy.
Hum Mol Genet
; 25(10): 1900-1911, 2016 05 15.
Artigo
Inglês
| MEDLINE | ID: mdl-26911675
6.
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes.
Mol Ther
; 25(4): 870-879, 2017 04 05.
Artigo
Inglês
| MEDLINE | ID: mdl-28279643
7.
A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.
Mol Ther
; 23(1): 192-201, 2015 Jan.
Artigo
Inglês
| MEDLINE | ID: mdl-25322757
8.
Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model.
Hum Mol Genet
; 22(24): 4929-37, 2013 Dec 15.
Artigo
Inglês
| MEDLINE | ID: mdl-23863459
9.
AAV1.NT-3 gene therapy for charcot-marie-tooth neuropathy.
Mol Ther
; 22(3): 511-521, 2014 Mar.
Artigo
Inglês
| MEDLINE | ID: mdl-24162799
10.
VIP-expressing dendritic cells protect against spontaneous autoimmune peripheral polyneuropathy.
Mol Ther
; 22(7): 1353-1363, 2014 Jul.
Artigo
Inglês
| MEDLINE | ID: mdl-24762627
11.
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
Inglês
| MEDLINE | ID: mdl-24145553
12.
Eteplirsen for the treatment of Duchenne muscular dystrophy.
Ann Neurol
; 74(5): 637-47, 2013 Nov.
Artigo
Inglês
| MEDLINE | ID: mdl-23907995
13.
Dystrophin immunity in Duchenne's muscular dystrophy.
N Engl J Med
; 363(15): 1429-37, 2010 Oct 07.
Artigo
Inglês
| MEDLINE | ID: mdl-20925545
14.
Molecular and clinical characterization of the myopathic form of mitochondrial DNA depletion syndrome caused by mutations in the thymidine kinase (TK2) gene.
Mol Genet Metab
; 110(1-2): 153-61, 2013.
Artigo
Inglês
| MEDLINE | ID: mdl-23932787
15.
Camptocormia as a late presentation in a manifesting carrier of duchenne muscular dystrophy.
Muscle Nerve
; 47(1): 124-7, 2013 Jan.
Artigo
Inglês
| MEDLINE | ID: mdl-23169483
16.
Impaired regeneration in LGMD2A supported by increased PAX7-positive satellite cell content and muscle-specific microrna dysregulation.
Muscle Nerve
; 47(5): 731-9, 2013 May.
Artigo
Inglês
| MEDLINE | ID: mdl-23553538
17.
Update on the treatment of Duchenne muscular dystrophy.
Curr Neurol Neurosci Rep
; 13(3): 332, 2013 Mar.
Artigo
Inglês
| MEDLINE | ID: mdl-23328943
18.
Dystrophin glycoprotein complex dysfunction: a regulatory link between muscular dystrophy and cancer cachexia.
Cancer Cell
; 8(5): 421-32, 2005 Nov.
Artigo
Inglês
| MEDLINE | ID: mdl-16286249
19.
AAV1.NT-3 gene therapy in the SOD1KO mouse model of accelerated sarcopenia.
J Cachexia Sarcopenia Muscle
; 14(5): 2204-2215, 2023 Oct.
Artigo
Inglês
| MEDLINE | ID: mdl-37553101
20.
AAV1.NT-3 gene therapy prevents age-related sarcopenia.
Aging (Albany NY)
; 15(5): 1306-1329, 2023 03 09.
Artigo
Inglês
| MEDLINE | ID: mdl-36897179