Detalhe da pesquisa
1.
Astrocytes influence the severity of spinal muscular atrophy.
Hum Mol Genet
; 24(14): 4094-102, 2015 Jul 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-25911676
2.
Defining the therapeutic window in a severe animal model of spinal muscular atrophy.
Hum Mol Genet
; 23(17): 4559-68, 2014 Sep 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-24722206
3.
Development and characterization of an SMN2-based intermediate mouse model of Spinal Muscular Atrophy.
Hum Mol Genet
; 22(9): 1843-55, 2013 May 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-23390132
4.
Restoration of SMN to Emx-1 expressing cortical neurons is not sufficient to provide benefit to a severe mouse model of Spinal Muscular Atrophy.
Transgenic Res
; 22(5): 1029-36, 2013 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-23512182
5.
Partial restoration of cardio-vascular defects in a rescued severe model of spinal muscular atrophy.
J Mol Cell Cardiol
; 52(5): 1074-82, 2012 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-22285962
6.
Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of spinal muscular atrophy.
Biochem Biophys Res Commun
; 417(1): 376-81, 2012 Jan 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-22172949
7.
Self-oligomerization regulates stability of survival motor neuron protein isoforms by sequestering an SCFSlmb degron.
Mol Biol Cell
; 29(2): 96-110, 2018 01 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-29167380
8.
Plastin-3 extends survival and reduces severity in mouse models of spinal muscular atrophy.
JCI Insight
; 2(5): e89970, 2017 03 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-28289706
9.
Decreasing disease severity in symptomatic, Smn(-/-);SMN2(+/+), spinal muscular atrophy mice following scAAV9-SMN delivery.
Hum Gene Ther
; 23(3): 330-5, 2012 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-22029744
10.
Delivery of therapeutic agents through intracerebroventricular (ICV) and intravenous (IV) injection in mice.
J Vis Exp
; (56)2011 Oct 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-21988897