Research graph
References from Autophagosome-tethering compounds selectively lower mutant ataxin-3 in preclinical models of spinocerebellar ataxia type 3. Local targets link to admitted publications; unresolved targets remain external evidence.
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10.1159/000358801 · External reference
Spinocerebellar ataxia. Nature reviews.
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Updated frequency analysis of spinocerebellar ataxia in China
10.1093/brain/awy016 · 2018 · External reference
Spinocerebellar ataxias: prospects and challenges for therapy development
10.1038/s41582-018-0051-6 · 2018 · External reference
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In vivo delivery of CRISPR-Cas9 genome editing components for therapeutic applications
10.1016/j.biomaterials.2022.121876 · 2022 · External reference
SnapShot: Targeted protein degradation
10.1016/j.cell.2024.10.025 · 2024 · External reference
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10.1080/15548627.2019.1688556 · External reference
Small Molecules Inducing Autophagic Degradation of Expanded Polyglutamine Protein through Interaction with Both Mutant ATXN3 and LC3
10.3390/ijms251910707 · 2024 · External reference
Gelation of cytoplasmic expanded CAG RNA repeats suppresses global protein synthesis
10.1038/s41589-023-01384-5 · 2023 · External reference
10.1016/j.ajhg.2024.03.015
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10.1093/hmg/11.9.1075 · External reference
Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models
10.1016/j.omtn.2017.04.005 · 2017 · External reference
10.1002/ana.25264
10.1002/ana.25264 · External reference
Silencing mutant ATXN3 expression resolves molecular phenotypes in SCA3 transgenic mice
10.1038/mt.2013.152 · 2013 · External reference
Antisense Oligonucleotide Therapy Targeted Against ATXN3 Improves Potassium Channel-Mediated Purkinje Neuron Dysfunction in Spinocerebellar Ataxia Type 3. Cerebellum (London
2021 · External reference
Allele-specific targeting of mutant ataxin-3 by antisense oligonucleotides in SCA3-iPSC-derived neurons
10.1016/j.omtn.2021.11.015 · 2022 · External reference
Selective autophagy as a potential therapeutic target for neurodegenerative disorders
10.1016/s1474-4422(18)30238-2 · 2018 · External reference
10.4161/auto.4600
10.4161/auto.4600 · External reference
10.1016/s0896-6273(00)80943-5
10.1016/s0896-6273(00)80943-5 · External reference
10.1007/978-3-319-71779-1_11
10.1007/978-3-319-71779-1_11 · External reference
Biochemical analysis to study wild-type and polyglutamine-expanded ATXN3 species
10.1371/journal.pone.0315868 · 2024 · External reference
10.1016/j.neuron.2013.05.029
10.1016/j.neuron.2013.05.029 · External reference
Haplotype-specific MAPK3 expression in 16p11.2 deletion contributes to variable neurodevelopment
10.1093/brain/awad071 · 2023 · External reference
