Chronic Enzyme Replacement to the Brain of a Late Infantile Neuronal Ceroid Lipofuscinosis Mouse Has Differential Effects on Phenotypes of Disease-Reference-Cited by-同舟云学术

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Chronic Enzyme Replacement to the Brain of a Late Infantile Neuronal Ceroid Lipofuscinosis Mouse Has Differential Effects on Phenotypes of Disease

Published:2017-03 Issue: Volume:4 Page:204-212
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Wiseman Jennifer A.,Meng Yu,Nemtsova Yuliya,Matteson Paul G.,Millonig James H.,Moore Dirk F.,Sleat David E.,Lobel Peter

Funder

NIH

Batten Disease Support and Research Association

Cures Within Reach

Noah’s Hope

Hope 4 Bridget

Jasper Against Batten

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference34 articles.

1. Association of mutations in a lysosomal protein with classical late-infantile neuronal ceroid lipofuscinosis;Sleat;Science,1997

2. The Neuronal Ceroid Lipofuscinoses (Batten Disease);Mole,2011

3. Treatment of lysosomal storage disorders: successes and challenges;Hollak;J. Inherit. Metab. Dis.,2014

4. Natural history of CLN2 disease: quantitative assessment of disease characteristics and rate of progression;Nickel;Neuropediatrics,2016

5. Intracerebroventricular Cerliponase Alfa (BMN 190) in children with CLN2 disease: interim results from a phase 1/2, open-label, dose-escalation study;Schulz;Neuropediatrics,2016

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1. A mouse mutant deficient in both neuronal ceroid lipofuscinosis‐associated proteins CLN3 and TPP1;Journal of Inherited Metabolic Disease;2023-04-26

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3. Brain transcriptome analysis of a CLN2 mouse model as a function of disease progression;Journal of Neuroinflammation;2021-11-08

4. Tween® Preserves Enzyme Activity and Stability in PLGA Nanoparticles;Nanomaterials;2021-11-03

5. Neuronal Ceroid Lipofuscinosis: Potential for Targeted Therapy;Drugs;2020-11-26

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