Detalhe da pesquisa
1.
Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR).
Ann Neurol
; 94(5): 955-968, 2023 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-37539981
2.
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
em Inglês
| MEDLINE | ID: mdl-37577753
3.
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mol Ther
; 29(2): 464-488, 2021 02 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33309881
4.
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.
N Engl J Med
; 377(18): 1713-1722, 2017 11 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-29091557
5.
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
6.
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
7.
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes.
Mol Ther
; 25(4): 870-879, 2017 04 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-28279643
8.
A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.
Mol Ther
; 23(1): 192-201, 2015 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-25322757
9.
Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model.
Hum Mol Genet
; 22(24): 4929-37, 2013 Dec 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-23863459
10.
AAV1.NT-3 gene therapy for charcot-marie-tooth neuropathy.
Mol Ther
; 22(3): 511-521, 2014 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-24162799
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
em Inglês
| MEDLINE | ID: mdl-24145553
12.
Eteplirsen for the treatment of Duchenne muscular dystrophy.
Ann Neurol
; 74(5): 637-47, 2013 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-23907995
13.
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
14.
Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74.
Mol Ther Methods Clin Dev
; 32(1): 101195, 2024 Mar 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-38327805
15.
Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results.
Nat Med
; 30(1): 199-206, 2024 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-38177855
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
em Inglês
| MEDLINE | ID: mdl-23553538
17.
Update on the treatment of Duchenne muscular dystrophy.
Curr Neurol Neurosci Rep
; 13(3): 332, 2013 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-23328943
18.
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
19.
Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States.
J Mark Access Health Policy
; 11(1): 2216518, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37261034
20.
Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model.
Mol Ther Methods Clin Dev
; 28: 284-299, 2023 Mar 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-36816759