Neuronal genetic rescue normalizes brain network dynamics in a lysosomal storage disorder despite persistent storage accumulation-Reference-Cited by-同舟云学术

Neuronal genetic rescue normalizes brain network dynamics in a lysosomal storage disorder despite persistent storage accumulation

Published:2022-07 Issue:7 Volume:30 Page:2464-2473
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Ahrens-Nicklas Rebecca C.,Tecedor Luis^ORCID,Hall Arron F.^ORCID,Kane Owen^ORCID,Chung Richard J.^ORCID,Lysenko Elena,Marsh Eric D.^ORCID,Stein Colleen S.,Davidson Beverly L.

Funder

NICHD

NINDS

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference43 articles.

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5. Combined anti-inflammatory and neuroprotective treatments have the potential to impact disease phenotypes in Cln3 (-/-) mice;Tarczyluk-Wells;Front. Neurol.,2019

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1. Upregulation of peroxisome proliferator-activated receptor γ with resorcinol alleviates reactive oxygen species generation and lipid accumulation in neuropathic lysosomal storage diseases;The International Journal of Biochemistry & Cell Biology;2024-09

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3. Cellular Modeling of CLN6 with IPSC-derived Neurons and Glia;2024-01-30

4. Protracted CLN3 Batten disease in mice that genetically model an exon-skipping therapeutic approach;Molecular Therapy - Nucleic Acids;2023-09

5. Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse model;Journal of Clinical Investigation;2023-06-15