Detalhe da pesquisa
1.
Confronting complexity in the design and conduct of high-risk rare disease clinical trials.
Mol Ther
; 31(5): 1191-1192, 2023 05 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-37141855
2.
Broader Implications of Progressive Liver Dysfunction and Lethal Sepsis in Two Boys following Systemic High-Dose AAV.
Mol Ther
; 28(8): 1753-1755, 2020 08 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-32710826
3.
Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.
Blood
; 115(23): 4678-88, 2010 Jun 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-20335222
4.
Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.
Mol Ther
; 18(7): 1318-29, 2010 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-20424599
5.
Myosin gene mutation correlates with anatomical changes in the human lineage.
Nature
; 428(6981): 415-8, 2004 Mar 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-15042088
6.
Safety and Efficacy of Regional Intravenous (RI) Versus Intramuscular (IM) Delivery of rAAV1 and rAAV8 to Nonhuman Primate Skeletal Muscle.
Mol Ther
; 16(7): 1291-1299, 2008 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-28178483
7.
Safety and efficacy of regional intravenous (r.i.) versus intramuscular (i.m.) delivery of rAAV1 and rAAV8 to nonhuman primate skeletal muscle.
Mol Ther
; 16(7): 1291-9, 2008 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-18461055
8.
Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models.
Nat Med
; 25(10): 1505-1511, 2019 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-31591596
9.
Signs of progress in gene therapy for muscular dystrophy also warrant caution.
Mol Ther
; 20(2): 249-51, 2012 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-22297820
10.
Mechanism of Deletion Removing All Dystrophin Exons in a Canine Model for DMD Implicates Concerted Evolution of X Chromosome Pseudogenes.
Mol Ther Methods Clin Dev
; 4: 62-71, 2017 Mar 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-28344992
11.
Suite of clinically relevant functional assays to address therapeutic efficacy and disease mechanism in the dystrophic mdx mouse.
J Appl Physiol (1985)
; 122(3): 593-602, 2017 Mar 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-27932677
12.
Uniform scale-independent gene transfer to striated muscle after transvenular extravasation of vector.
Circulation
; 112(12): 1780-8, 2005 Sep 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-16157771
13.
Limb-girdle muscular dystrophy in the United States.
J Neuropathol Exp Neurol
; 65(10): 995-1003, 2006 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-17021404
14.
Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs.
Hum Gene Ther Clin Dev
; 26(1): 5-14, 2015 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-25675273
15.
Perspectives on best practices for gene therapy programs.
Hum Gene Ther
; 26(3): 127-33, 2015 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-25654329
16.
Sarcomeres are added in series to emphysematous rat diaphragm after lung volume reduction surgery.
Chest
; 121(1): 210-5, 2002 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-11796453
17.
Myosin heavy chain and physiological adaptation of the rat diaphragm in elastase-induced emphysema.
Respir Res
; 4: 1, 2003.
Artigo
em Inglês
| MEDLINE | ID: mdl-12617755
18.
Inspiratory loading does not accelerate dystrophy in mdx mouse diaphragm: implications for regenerative therapy.
J Appl Physiol (1985)
; 94(2): 411-9, 2003 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-12531909
19.
Global cardiac-specific transgene expression using cardiopulmonary bypass with cardiac isolation.
Ann Thorac Surg
; 73(6): 1939-46, 2002 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-12078794
20.
Cardiac surgical delivery of the sarcoplasmic reticulum calcium ATPase rescues myocytes in ischemic heart failure.
Ann Thorac Surg
; 96(2): 586-95, 2013 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-23773730