CNS-directed AAV2-mediated gene therapy ameliorates functional deficits in a murine model of infantile neuronal ceroid lipofuscinosis.
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Glycosylation, transport, and complex formation of palmitoyl protein thioesterase 1 (PPT1)--distinct characteristics in neuronsMoving towards effective therapeutic strategies for Neuronal Ceroid LipofuscinosisThe novel Cln1(R151X) mouse model of infantile neuronal ceroid lipofuscinosis (INCL) for testing nonsense suppression therapyInteractions of the proteins of neuronal ceroid lipofuscinosis: clues to functionComprehensive functional characterization of murine infantile Batten disease including Parkinson-like behavior and dopaminergic markers.Purkinje cell-specific males absent on the first (mMof) gene deletion results in an ataxia-telangiectasia-like neurological phenotype and backward walking in mice.Excitatory input onto hilar somatostatin interneurons is increased in a chronic model of epilepsyNeuroprotection and lifespan extension in Ppt1(-/-) mice by NtBuHA: therapeutic implications for INCLRapamycin prevents epilepsy in a mouse model of tuberous sclerosis complex.Gene expression profiling in a mouse model of infantile neuronal ceroid lipofuscinosis reveals upregulation of immediate early genes and mediators of the inflammatory responseTreatment of the Ppt1(-/-) mouse model of infantile neuronal ceroid lipofuscinosis with the N-methyl-D-aspartate (NMDA) receptor antagonist memantine.Efficient intrathymic gene transfer following in situ administration of a rAAV serotype 8 vector in mice and nonhuman primates.Human recombinant palmitoyl-protein thioesterase-1 (PPT1) for preclinical evaluation of enzyme replacement therapy for infantile neuronal ceroid lipofuscinosis.Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis.A mutation in canine PPT1 causes early onset neuronal ceroid lipofuscinosis in a Dachshund.An anti-neuroinflammatory that targets dysregulated glia enhances the efficacy of CNS-directed gene therapy in murine infantile neuronal ceroid lipofuscinosisAdeno-associated virus-mediated gene transferNeurologic abnormalities in mouse models of the lysosomal storage disorders mucolipidosis II and mucolipidosis III γ.Pathogenesis and therapies for infantile neuronal ceroid lipofuscinosis (infantile CLN1 disease).Lentiviral-mediated gene transfer to the sheep brain: implications for gene therapy in Batten disease.The role of attenuated astrocyte activation in infantile neuronal ceroid lipofuscinosis.Long-term Effects of Multiple Glucocorticoid Exposures in Neonatal MiceSuccessive neuron loss in the thalamus and cortex in a mouse model of infantile neuronal ceroid lipofuscinosis.Reversible Cysteine Acylation Regulates the Activity of Human Palmitoyl-Protein Thioesterase 1 (PPT1).CNS-directed gene therapy for lysosomal storage diseasesSynergistic effects of central nervous system-directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosisIntravenous 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.Tissue-specific variation in nonsense mutant transcript level and drug-induced read-through efficiency in the Cln1(R151X) mouse model of INCLEfficacy of nonviral gene transfer in the canine brain.Therapeutic approaches to the challenge of neuronal ceroid lipofuscinoses.Cerebellar pathology and motor deficits in the palmitoyl protein thioesterase 1-deficient mouseA murine model of infantile neuronal ceroid lipofuscinosis-ultrastructural evaluation of storage in the central nervous system and viscera.The natural history and treatment of epilepsy in a murine model of tuberous sclerosis.Considerations for the treatment of infantile neuronal ceroid lipofuscinosis (infantile Batten disease)Gene therapy for the neurological manifestations in lysosomal storage disorders.Synergistic effects of treating the spinal cord and brain in CLN1 disease.Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.Altered glutamate receptor function in the cerebellum of the Ppt1-/- mouse, a murine model of infantile neuronal ceroid lipofuscinosisUsing Patient-Specific Induced Pluripotent Stem Cells and Wild-Type Mice to Develop a Gene Augmentation-Based Strategy to Treat CLN3-Associated Retinal DegenerationSurvival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis.
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P2860
CNS-directed AAV2-mediated gene therapy ameliorates functional deficits in a murine model of infantile neuronal ceroid lipofuscinosis.
description
2005 nî lūn-bûn
@nan
2005年の論文
@ja
2005年学术文章
@wuu
2005年学术文章
@zh
2005年学术文章
@zh-cn
2005年学术文章
@zh-hans
2005年学术文章
@zh-my
2005年学术文章
@zh-sg
2005年學術文章
@yue
2005年學術文章
@zh-hant
name
CNS-directed AAV2-mediated gen ...... euronal ceroid lipofuscinosis.
@en
CNS-directed AAV2-mediated gen ...... euronal ceroid lipofuscinosis.
@nl
type
label
CNS-directed AAV2-mediated gen ...... euronal ceroid lipofuscinosis.
@en
CNS-directed AAV2-mediated gen ...... euronal ceroid lipofuscinosis.
@nl
prefLabel
CNS-directed AAV2-mediated gen ...... euronal ceroid lipofuscinosis.
@en
CNS-directed AAV2-mediated gen ...... euronal ceroid lipofuscinosis.
@nl
P2093
P50
P1433
P1476
CNS-directed AAV2-mediated gen ...... neuronal ceroid lipofuscinosis
@en
P2093
David Wozniak
Ellen Bible
Kendra Johnson
Megan A Griffey
Michael Wong
Steven M Rothman
P304
P356
10.1016/J.YMTHE.2005.11.008
P577
2005-12-20T00:00:00Z