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.
Identifying Biomarkers of Spinal Muscular Atrophy for Further Development.
J Neuromuscul Dis
; 10(5): 937-954, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37458045
6.
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
7.
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
8.
Treatment Algorithm for Infants Diagnosed with Spinal Muscular Atrophy through Newborn Screening.
J Neuromuscul Dis
; 5(2): 145-158, 2018.
Artigo
em Inglês
| MEDLINE | ID: mdl-29614695
9.
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
10.
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
11.
The RNA-Binding Protein HuR Posttranscriptionally Regulates IL-2 Homeostasis and CD4+ Th2 Differentiation.
Immunohorizons
; 1(6): 109-123, 2017 Aug 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-30035254
12.
Revised Recommendations for the Treatment of Infants Diagnosed with Spinal Muscular Atrophy Via Newborn Screening Who Have 4 Copies of SMN2.
J Neuromuscul Dis
; 7(2): 97-100, 2020.
Artigo
em Inglês
| MEDLINE | ID: mdl-32007960
13.
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
14.
Combination of SMN trans-splicing and a neurotrophic factor increases the life span and body mass in a severe model of spinal muscular atrophy.
Hum Gene Ther
; 22(2): 135-44, 2011 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-20804424
15.
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