Enzyme replacement therapy attenuates disease progression in a canine model of late-infantile neuronal ceroid lipofuscinosis (CLN2 disease).
about
Moving towards effective therapeutic strategies for Neuronal Ceroid LipofuscinosisAAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease.Extraneuronal pathology in a canine model of CLN2 neuronal ceroid lipofuscinosis after intracerebroventricular gene therapy that delays neurological disease progression.Safety Evaluation of CNS Administered Biologics-Study Design, Data Interpretation, and Translation to the Clinic.Safety and potential efficacy of gemfibrozil as a supportive treatment for children with late infantile neuronal ceroid lipofuscinosis and other lipid storage disorders.A rare homozygous MFSD8 single-base-pair deletion and frameshift in the whole genome sequence of a Chinese Crested dog with neuronal ceroid lipofuscinosis.Multifocal retinopathy in Dachshunds with CLN2 neuronal ceroid lipofuscinosis.Intracerebroventricular gene therapy that delays neurological disease progression is associated with selective preservation of retinal ganglion cells in a canine model of CLN2 disease.Australian Cattle Dogs with Neuronal Ceroid Lipofuscinosis are Homozygous for a CLN5 Nonsense Mutation Previously Identified in Border Collies.Disease models for the development of therapies for lysosomal storage diseases.Chronic Enzyme Replacement to the Brain of a Late Infantile Neuronal Ceroid Lipofuscinosis Mouse Has Differential Effects on Phenotypes of Disease.Synergistic effects of treating the spinal cord and brain in CLN1 disease.A canine model for neuronal ceroid lipofuscinosis highlights the promise of gene therapy for lysosomal storage diseases.Canine neuronal ceroid lipofuscinoses: Promising models for preclinical testing of therapeutic interventions.AAVrh.10-mediated APOE2 CNS Gene Therapy for APOE4-associated Alzheimer's Disease.Unique Characteristics of the Photoparoxysmal Response in Patients With Neuronal Ceroid Lipofuscinosis Type 2: Can EEG Be a Biomarker?Pharmacokinetics and dynamics of mycophenolate mofetil after single-dose oral administration in juvenile dachshunds.Longitudinal In Vivo Monitoring of the CNS Demonstrates the Efficacy of Gene Therapy in a Sheep Model of CLN5 Batten Disease
P2860
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P2860
Enzyme replacement therapy attenuates disease progression in a canine model of late-infantile neuronal ceroid lipofuscinosis (CLN2 disease).
description
2014 nî lūn-bûn
@nan
2014 թուականի Յունիսին հրատարակուած գիտական յօդուած
@hyw
2014 թվականի հունիսին հրատարակված գիտական հոդված
@hy
2014年の論文
@ja
2014年論文
@yue
2014年論文
@zh-hant
2014年論文
@zh-hk
2014年論文
@zh-mo
2014年論文
@zh-tw
2014年论文
@wuu
name
Enzyme replacement therapy att ...... lipofuscinosis (CLN2 disease).
@ast
Enzyme replacement therapy att ...... lipofuscinosis (CLN2 disease).
@en
Enzyme replacement therapy att ...... neuronal ceroid lipofuscinosis
@nl
type
label
Enzyme replacement therapy att ...... lipofuscinosis (CLN2 disease).
@ast
Enzyme replacement therapy att ...... lipofuscinosis (CLN2 disease).
@en
Enzyme replacement therapy att ...... neuronal ceroid lipofuscinosis
@nl
prefLabel
Enzyme replacement therapy att ...... lipofuscinosis (CLN2 disease).
@ast
Enzyme replacement therapy att ...... lipofuscinosis (CLN2 disease).
@en
Enzyme replacement therapy att ...... neuronal ceroid lipofuscinosis
@nl
P2093
P2860
P356
P1476
Enzyme replacement therapy att ...... lipofuscinosis (CLN2 disease).
@en
P2093
Brian R Vuillemenot
Charles A O'Neill
Christine M Sibigtroth
Derek Kennedy
Joan R Coates
Martin L Katz
Melissa Carpentier
Whitney M Young
P2860
P304
P356
10.1002/JNR.23423
P577
2014-06-17T00:00:00Z