Novel Drug Candidates Improve Ganglioside Accumulation and Neural Dysfunction in GM1 Gangliosidosis Models with Autophagy Activation-Reference-Cited by-同舟云学术

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Novel Drug Candidates Improve Ganglioside Accumulation and Neural Dysfunction in GM1 Gangliosidosis Models with Autophagy Activation

Published:2020-05 Issue:5 Volume:14 Page:909-923
ISSN:2213-6711
Container-title:Stem Cell Reports
language:en
Short-container-title:Stem Cell Reports

Author:

Kajihara Ryutaro,Numakawa Tadahiro,Odaka Haruki,Yaginuma Yuji,Fusaki Noemi,Okumiya Toshika,Furuya Hirokazu,Inui Seiji,Era Takumi

Funder

Ministry of Health, Labour and Welfare of Japan

Japan Agency for Medical Research and Development

Japan Society for the Promotion of Science

Publisher

Elsevier BV

Subject

Cell Biology,Developmental Biology,Genetics,Biochemistry

Reference38 articles.

1. AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival;Baek;PLoS One,2010

2. A fluorescence-microscopic and cytofluorometric system for monitoring the turnover of the autophagic substrate p62/SQSTM1;Ben Younes;Autophagy,2011

3. Lysosomal multienzyme complex: pros and cons of working together;Bonten;Cell. Mol. Life Sci.,2014

4. GM(1) gangliosidosis: review of clinical, molecular, and therapeutic aspects;Brunetti-Pierri;Mol. Genet. Metab.,2008

5. Beneficial effects of substrate reduction therapy in a mouse model of GM1 gangliosidosis;Elliot-Smith;Mol. Genet. Metab.,2008

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1. Regulation of autophagy by ST3GAL2-mediated α2-3 sialylated glycosphingolipids in hepatic encephalopathy;International Journal of Biological Macromolecules;2024-10

2. Insights into the Pathobiology of GM1 Gangliosidosis from Single-Nucleus Transcriptomic Analysis of CNS Cells in a Mouse Model;International Journal of Molecular Sciences;2024-09-08

3. Creation of an in vitro model of GM1 gangliosidosis by CRISPR/Cas9 knocking‐out the GLB1 gene in SH‐SY5Y human neuronal cell line;Cell Biochemistry and Function;2024-07-30

4. Polymer-based drug delivery systems under investigation for enzyme replacement and other therapies of lysosomal storage disorders;Advanced Drug Delivery Reviews;2023-06

5. Integrated transcriptomics, proteomics and metabolomics to identify biomarkers of astragaloside IV against cerebral ischemic injury in rats;Food & Function;2023

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