Large-volume intrathecal enzyme delivery increases survival of a mouse model of late infantile neuronal ceroid lipofuscinosis.
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Moving towards effective therapeutic strategies for Neuronal Ceroid LipofuscinosisSialic acid deposition impairs the utility of AAV9, but not peptide-modified AAVs for brain gene therapy in a mouse model of lysosomal storage diseaseEffective intravenous therapy for neurodegenerative disease with a therapeutic enzyme and a peptide that mediates delivery to the brainSafety 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.Systemic administration of tripeptidyl peptidase I in a mouse model of late infantile neuronal ceroid lipofuscinosis: effect of glycan modification.Pathogenesis and therapies for infantile neuronal ceroid lipofuscinosis (infantile CLN1 disease).Biochemical evidence for superior correction of neuronal storage by chemically modified enzyme in murine mucopolysaccharidosis VII.Gemfibrozil and fenofibrate, Food and Drug Administration-approved lipid-lowering drugs, up-regulate tripeptidyl-peptidase 1 in brain cells via peroxisome proliferator-activated receptor α: implications for late infantile Batten disease therapy.Photosensitivity is an early marker of neuronal ceroid lipofuscinosis type 2 disease.Considerations for the treatment of infantile neuronal ceroid lipofuscinosis (infantile Batten disease)Chronic Enzyme Replacement to the Brain of a Late Infantile Neuronal Ceroid Lipofuscinosis Mouse Has Differential Effects on Phenotypes of Disease.Physiology of the intrathecal bolus: the leptomeningeal route for macromolecule and particle delivery to CNS.A Basic ApoE-Based Peptide Mediator to Deliver Proteins across the Blood-Brain Barrier: Long-Term Efficacy, Toxicity, and Mechanism.Proteomic Analysis of Brain and Cerebrospinal Fluid from the Three Major Forms of Neuronal Ceroid Lipofuscinosis Reveals Potential Biomarkers.Inducible transgenic expression of tripeptidyl peptidase 1 in a mouse model of late-infantile neuronal ceroid lipofuscinosis.Delivering drugs to the central nervous system: an overview.Flupirtine derivatives as potential treatment for the neuronal ceroid lipofuscinoses
P2860
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P2860
Large-volume intrathecal enzyme delivery increases survival of a mouse model of late infantile neuronal ceroid lipofuscinosis.
description
2011 nî lūn-bûn
@nan
2011 թուականի Յուլիսին հրատարակուած գիտական յօդուած
@hyw
2011 թվականի հուլիսին հրատարակված գիտական հոդված
@hy
2011年の論文
@ja
2011年論文
@yue
2011年論文
@zh-hant
2011年論文
@zh-hk
2011年論文
@zh-mo
2011年論文
@zh-tw
2011年论文
@wuu
name
Large-volume intrathecal enzym ...... euronal ceroid lipofuscinosis.
@ast
Large-volume intrathecal enzym ...... euronal ceroid lipofuscinosis.
@en
type
label
Large-volume intrathecal enzym ...... euronal ceroid lipofuscinosis.
@ast
Large-volume intrathecal enzym ...... euronal ceroid lipofuscinosis.
@en
prefLabel
Large-volume intrathecal enzym ...... euronal ceroid lipofuscinosis.
@ast
Large-volume intrathecal enzym ...... euronal ceroid lipofuscinosis.
@en
P2093
P2860
P356
P1433
P1476
Large-volume intrathecal enzym ...... euronal ceroid lipofuscinosis.
@en
P2093
David E Sleat
Istvan Sohar
Lingling Wang
Mukarram El-Banna
Peter Lobel
P2860
P304
P356
10.1038/MT.2011.130
P577
2011-07-05T00:00:00Z