Effective Intravenous Therapy for Neurodegenerative Disease With a Therapeutic Enzyme and a Peptide That Mediates Delivery to the Brain-Reference-Cited by-同舟云学术

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Effective Intravenous Therapy for Neurodegenerative Disease With a Therapeutic Enzyme and a Peptide That Mediates Delivery to the Brain

Published:2014-03 Issue:3 Volume:22 Page:547-553
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Meng Yu,Sohar Istvan,Sleat David E,Richardson Jason R,Reuhl Kenneth R,Jenkins Robert B,Sarkar Gobinda,Lobel Peter

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,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. Mitochondrial ATP synthase subunit c storage in the ceroid-lipofuscinoses (Batten disease);Palmer;Am J Med Genet,1992

3. The birth prevalence of lysosomal storage disorders in the Czech Republic: comparison with data in different populations;Poupetová;J Inherit Metab Dis,2010

4. Molecular epidemiology of childhood neuronal ceroid-lipofuscinosis in Italy;Santorelli;Orphanet J Rare Dis,2013

5. Enzyme replacement therapy for lysosomal diseases: lessons from 20 years of experience and remaining challenges;Desnick;Annu Rev Genomics Hum Genet,2012

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2. Advances in therapies for neurological lysosomal storage disorders;Journal of Inherited Metabolic Disease;2023-05-02

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

4. Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier;Molecular Therapy;2023-03

5. Elevated levels of tripeptidyl peptidase 1 do not ameliorate pathogenesis in a mouse model of Alzheimer disease;Neurobiology of Aging;2022-10

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