Detalhe da pesquisa
1.
A large animal model of spinal muscular atrophy and correction of phenotype.
Ann Neurol
; 77(3): 399-414, 2015 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-25516063
2.
Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.
Mol Ther
; 23(3): 477-87, 2015 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-25358252
3.
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
4.
Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS.
Mol Ther
; 21(12): 2148-59, 2013 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-24008656
5.
Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery.
Hum Mol Genet
; 19(20): 3895-905, 2010 Oct 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-20639395
6.
Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders.
Mol Ther
; 19(11): 1971-80, 2011 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-21811247
7.
Electrophysiological Biomarkers in Spinal Muscular Atrophy: Preclinical Proof of Concept.
Ann Clin Transl Neurol
; 1(1): 34-44, 2014 Jan 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-24511555
8.
Microglia induce motor neuron death via the classical NF-κB pathway in amyotrophic lateral sclerosis.
Neuron
; 81(5): 1009-1023, 2014 Mar 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-24607225