Detalhe da pesquisa
1.
Lack of germline transmission in male mice following a single intravenous administration of AAV5-hFVIII-SQ gene therapy.
Gene Ther
; 30(7-8): 581-586, 2023 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-35132205
2.
Safety Findings of Dosing Gene Therapy Vectors in NHP With Pre-existing or Treatment-Emergent Anti-capsid Antibodies.
Toxicol Pathol
; 51(5): 246-256, 2023 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-37921115
3.
Ex Vivo Gene Therapy: Graft-versus-host Disease (GVHD) in NSG™ (NOD.Cg-Prkdcscid Il2rgtm1Wjl/SzJ) Mice Transplanted with CD34+ Human Hematopoietic Stem Cells.
Toxicol Pathol
; 47(5): 656-660, 2019 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-31064282
4.
CNS Adverse Effects: From Functional Observation Battery/Irwin Tests to Electrophysiology.
Handb Exp Pharmacol
; 229: 83-113, 2015.
Artigo
em Inglês
| MEDLINE | ID: mdl-26091637
5.
AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Friedreich's ataxia.
Mol Ther Methods Clin Dev
; 32(1): 101193, 2024 Mar 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-38352270
6.
Cerebellar Pathology in an Inducible Mouse Model of Friedreich Ataxia.
Front Neurosci
; 16: 819569, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-35401081
7.
Muscle-directed gene therapy corrects Pompe disease and uncovers species-specific GAA immunogenicity.
EMBO Mol Med
; 14(1): e13968, 2022 01 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-34850579
8.
Young mice administered adult doses of AAV5-hFVIII-SQ achieve therapeutic factor VIII expression into adulthood.
Mol Ther Methods Clin Dev
; 26: 519-531, 2022 Sep 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-36092364
9.
Alpha4* nicotinic receptors in preBotzinger complex mediate cholinergic/nicotinic modulation of respiratory rhythm.
J Neurosci
; 28(2): 519-28, 2008 Jan 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-18184794
10.
Demonstration of functional alpha4-containing nicotinic receptors in the medial habenula.
Neuropharmacology
; 56(1): 247-53, 2009 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-18789953
11.
The Impact of Pre-existing Immunity on the Non-clinical Pharmacodynamics of AAV5-Based Gene Therapy.
Mol Ther Methods Clin Dev
; 13: 440-452, 2019 Jun 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-31193016
12.
Nicotine-induced dystonic arousal complex in a mouse line harboring a human autosomal-dominant nocturnal frontal lobe epilepsy mutation.
J Neurosci
; 27(38): 10128-42, 2007 Sep 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-17881519
13.
Novel seizure phenotype and sleep disruptions in knock-in mice with hypersensitive alpha 4* nicotinic receptors.
J Neurosci
; 25(49): 11396-411, 2005 Dec 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-16339034
14.
Increased sensitivity to agonist-induced seizures, straub tail, and hippocampal theta rhythm in knock-in mice carrying hypersensitive alpha 4 nicotinic receptors.
J Neurosci
; 23(7): 2582-90, 2003 Apr 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-12684443
15.
Knockin mice with Leu9'Ser alpha4-nicotinic receptors: substantia nigra dopaminergic neurons are hypersensitive to agonist and lost postnatally.
Physiol Genomics
; 18(3): 299-307, 2004 Aug 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-15199190
16.
Hypersensitive knockin mouse strains identify receptors and pathways for nicotine action.
Curr Opin Drug Discov Devel
; 6(5): 633-9, 2003 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-14579512
17.
Rapid reduction of ATP synthesis and lack of free radical formation by MPP+ in rat brain synaptosomes and mitochondria.
Brain Res
; 975(1-2): 214-21, 2003 Jun 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-12763610
18.
Mice expressing the ADNFLE valine 287 leucine mutation of the Β2 nicotinic acetylcholine receptor subunit display increased sensitivity to acute nicotine administration and altered presynaptic nicotinic receptor function.
Pharmacol Biochem Behav
; 103(3): 603-21, 2013 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-23123803
19.
Human mesenchymal stromal cells and their derivative, SB623 cells, rescue neural cells via trophic support following in vitro ischemia.
Cell Transplant
; 19(8): 973-84, 2010.
Artigo
em Inglês
| MEDLINE | ID: mdl-20350349
20.
Combining radio telemetry and automated blood sampling: a novel approach for integrative pharmacology and toxicology studies.
J Pharmacol Toxicol Methods
; 62(1): 30-9, 2010.
Artigo
em Inglês
| MEDLINE | ID: mdl-20507819