Detalhe da pesquisa
1.
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.
N Engl J Med
; 377(18): 1713-1722, 2017 11 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-29091557
2.
Oligodendrocytes contribute to motor neuron death in ALS via SOD1-dependent mechanism.
Proc Natl Acad Sci U S A
; 113(42): E6496-E6505, 2016 10 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-27688759
3.
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
4.
A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.
Mol Ther
; 23(1): 192-201, 2015 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-25322757
5.
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
6.
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
7.
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
8.
Additive amelioration of ALS by co-targeting independent pathogenic mechanisms.
Ann Clin Transl Neurol
; 4(2): 76-86, 2017 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-28168207
9.
Retraction Note: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN.
Nat Biotechnol
; 40(11): 1692, 2022 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-36203015
10.
Adeno Associated Virus 9-Based Gene Therapy Delivers a Functional Monocarboxylate Transporter 8, Improving Thyroid Hormone Availability to the Brain of Mct8-Deficient Mice.
Thyroid
; 26(9): 1311-9, 2016 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-27432638
11.
Major histocompatibility complex class I molecules protect motor neurons from astrocyte-induced toxicity in amyotrophic lateral sclerosis.
Nat Med
; 22(4): 397-403, 2016 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-26928464
12.
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
13.
Aging brain microenvironment decreases hippocampal neurogenesis through Wnt-mediated survivin signaling.
Aging Cell
; 11(3): 542-52, 2012 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-22404871
14.
Astrocytes from familial and sporadic ALS patients are toxic to motor neurons.
Nat Biotechnol
; 29(9): 824-8, 2011 Aug 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-21832997
15.
Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN.
Nat Biotechnol
; 28(3): 271-4, 2010 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-20190738