Detalhe da pesquisa
1.
Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late-gestation nonhuman primates.
FASEB J
; 29(9): 3876-88, 2015 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-26062602
2.
An anti-neuroinflammatory that targets dysregulated glia enhances the efficacy of CNS-directed gene therapy in murine infantile neuronal ceroid lipofuscinosis.
J Neurosci
; 34(39): 13077-82, 2014 Sep 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-25253854
3.
Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis.
Mol Genet Metab
; 114(2): 281-93, 2015 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-25257657
4.
Early glial activation, synaptic changes and axonal pathology in the thalamocortical system of Niemann-Pick type C1 mice.
Neurobiol Dis
; 45(3): 1086-100, 2012 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-22198570
5.
Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl-protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis.
Mol Genet Metab
; 107(1-2): 213-21, 2012 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-22704978
6.
Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system.
FASEB J
; 25(10): 3505-18, 2011 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-21746868
7.
DHHC5 interacts with PDZ domain 3 of post-synaptic density-95 (PSD-95) protein and plays a role in learning and memory.
J Biol Chem
; 285(17): 13022-31, 2010 Apr 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-20178993
8.
Current therapies for the soluble lysosomal forms of neuronal ceroid lipofuscinosis.
Biochem Soc Trans
; 38(6): 1484-8, 2010 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-21118112
9.
In utero gene transfer to the mouse nervous system.
Biochem Soc Trans
; 38(6): 1489-93, 2010 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-21118113
10.
Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors.
Sci Rep
; 10(1): 2121, 2020 02 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-32034258
11.
Fetal gene therapy for neurodegenerative disease of infants.
Nat Med
; 24(9): 1317-1323, 2018 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-30013199
12.
Proteomic mapping of differentially vulnerable pre-synaptic populations identifies regulators of neuronal stability in vivo.
Sci Rep
; 7(1): 12412, 2017 09 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-28963550
13.
Non-invasive molecular imaging of inflammatory macrophages in allograft rejection.
EJNMMI Res
; 5(1): 69, 2015 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-26611870
14.
Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 gene.
Hum Gene Ther
; 25(3): 223-39, 2014 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-24372003
15.
Behavioral deficits, early gliosis, dysmyelination and synaptic dysfunction in a mouse model of mucolipidosis IV.
Acta Neuropathol Commun
; 2: 133, 2014 Sep 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-25200117
16.
Regional brain atrophy in mouse models of neuronal ceroid lipofuscinosis: a new rostrocaudal perspective.
J Child Neurol
; 28(9): 1117-22, 2013 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-24014506
17.
Disruption of the autophagy-lysosome pathway is involved in neuropathology of the nclf mouse model of neuronal ceroid lipofuscinosis.
PLoS One
; 7(4): e35493, 2012.
Artigo
em Inglês
| MEDLINE | ID: mdl-22536393