Detalhe da pesquisa
1.
Motor unit recovery following Smn restoration in mouse models of spinal muscular atrophy.
Hum Mol Genet
; 31(18): 3107-3119, 2022 09 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-35551393
2.
Comparison of independent screens on differentially vulnerable motor neurons reveals alpha-synuclein as a common modifier in motor neuron diseases.
PLoS Genet
; 13(3): e1006680, 2017 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-28362802
3.
Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy.
Mol Ther
; 24(9): 1592-601, 2016 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-27401142
4.
Rescue of a Mouse Model of Spinal Muscular Atrophy With Respiratory Distress Type 1 by AAV9-IGHMBP2 Is Dose Dependent.
Mol Ther
; 24(5): 855-66, 2016 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-26860981
5.
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
6.
Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models.
Hum Mol Genet
; 23(18): 4832-45, 2014 Sep 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-24781211
7.
Placental development in a mouse model of spinal muscular atrophy.
Biochem Biophys Res Commun
; 470(1): 82-87, 2016 Jan 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-26748185
8.
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
9.
Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophy.
Mol Ther
; 20(1): 119-26, 2012 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-22031236
10.
Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy.
Hum Mol Genet
; 18(9): 1600-11, 2009 May 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-19228773
11.
Short-duration splice promoting compound enables a tunable mouse model of spinal muscular atrophy.
Life Sci Alliance
; 4(1)2021 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-33234679
12.
Minor snRNA gene delivery improves the loss of proprioceptive synapses on SMA motor neurons.
JCI Insight
; 5(12)2020 06 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-32516136
13.
Functional characterization of SMN evolution in mouse models of SMA.
Sci Rep
; 9(1): 9472, 2019 07 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31263170
14.
Analysis of Azithromycin Monohydrate as a Single or a Combinatorial Therapy in a Mouse Model of Severe Spinal Muscular Atrophy.
J Neuromuscul Dis
; 4(3): 237-249, 2017.
Artigo
em Inglês
| MEDLINE | ID: mdl-28598854
15.
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
16.
Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compounds.
EMBO Mol Med
; 5(7): 1103-18, 2013 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-23740718
17.
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
18.
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