ArticleProceedings of the National Academy of Sciences of the United States of America2020
Translation of the intrinsically disordered protein α-synuclein is inhibited by a small molecule targeting its structured mRNA.
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Article in Proceedings of the National Academy of Sciences of the United States of America, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 78 papers, 1 of them a synthesis that pooled it.
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78citing papers in PubMed, 1 pooled it
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Who cites it
78 citing papers in PubMed, 1 synthesis or guideline pooled it, 121 citations in OpenAlex.
- RNA Drugs and RNA Targets for Small Molecules: Principles, Progress, and Challenges.Pharmacological reviews · 2020Pooled it
- From synaptic development to degeneration: a narrative review of small molecule strategies targeting alpha-synuclein in Parkinson's disease.Metabolic brain disease · 2026Review
- Gaps in the Parkinson's disease therapeutic clinical pipeline: A focus on approaches targeting disease pathobiology.Journal of Parkinson's disease · 2026Review
- Macrophage ferritin heavy chain/α-synuclein regulatory axis modulates ferroptosis during kidney injury.JCI insight · 2026Article
- Comparative analysis of alkyne- and desthiobiotinylated photoaffinity probes for chemotranscriptomic profiling.RSC chemical biology · 2026Article
- Targeting RNA binding proteins with small-molecule inhibitors: Advances, challenges, and therapeutic opportunities.Acta pharmaceutica Sinica. B · 2026Review
- Predicting small molecule-RNA interactions without RNA tertiary structures.Nature biotechnology · 2026Article
- Targeting G-Rich lncRNA and Its Structural Polymorphism With Selective G-Quadruplex Ligands: An NMR Study.Chemistry (Weinheim an der Bergstrasse, Germany) · 2025Article
- Cardiolipin and mitochondrial membrane integrity in neurodegeneration: insights from α-synuclein-driven Parkinson's disease.Acta neuropathologica communications · 2025Review
- Structure-Guided Design of a Bioactive Covalent Small Molecule Targeting a Riboswitch.Journal of the American Chemical Society · 2025Article
- Discovery of Macrocyclic Peptide Binders, Covalent Modifiers, and Degraders of a Structured RNA by mRNA Display.Journal of the American Chemical Society · 2025Article
- Review
- Aryl Guanyl Hydrazones: A Viable Strategy for Designing BBB-Permeable, Neuroactive Compounds?ACS chemical neuroscience · 2025Review
- Mechanistic studies of small molecule ligands selective to RNA single G bulges.Nucleic acids research · 2025Article
- Exploring α-Syn's Functions Through Ablation Models: Physiological and Pathological Implications.Cellular and molecular neurobiology · 2025Review
- The Unified Theory of Neurodegeneration Pathogenesis Based on Axon Deamidation.International journal of molecular sciences · 2025Review
- The Druggable Transcriptome Project: From Chemical Probes to Precision Medicines.Biochemistry · 2025Review
- Article
- Alpha-Synuclein Pathophysiology in Neurodegenerative Disorders: A Review Focusing on Molecular Mechanisms and Treatment Advances in Parkinson's Disease.Cellular and molecular neurobiology · 2025Review
- Tailored Intranasal Albumin Caged Selegiline-α Synuclein siRNA Liposome with Improved Efficiency in Parkinson's Model.Pharmaceutics · 2025Article
18 more citing papers are in PubMed but not listed here.
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Authors and funding
14 authors at 3 institutions in 1 country.
Peiyuan ZhangDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458.ORCID 0000-0003-4086-3436
Hye-Jin ParkRutgers Robert Wood Johnson Medical School Institute for Neurological Therapeutics, Rutgers Robert Wood Johnson Medical School, Piscataway, NJ 08854.
Jie ZhangRutgers Robert Wood Johnson Medical School Institute for Neurological Therapeutics, Rutgers Robert Wood Johnson Medical School, Piscataway, NJ 08854.
Eunsung JunnRutgers Robert Wood Johnson Medical School Institute for Neurological Therapeutics, Rutgers Robert Wood Johnson Medical School, Piscataway, NJ 08854.
Ryan J AndrewsRoy J. Carver Department of Biophysics, Biochemistry and Molecular Biology, Iowa State University, Ames, IA 50011.
Sai Pradeep VelagapudiDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458.
Daniel AbeggDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458.
Kamalakannan VishnuDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458.
Matthew G CostalesDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458.
Jessica L Childs-DisneyDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458.
Alexander AdibekianDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458.
Walter N MossRoy J. Carver Department of Biophysics, Biochemistry and Molecular Biology, Iowa State University, Ames, IA 50011.
M Maral MouradianRutgers Robert Wood Johnson Medical School Institute for Neurological Therapeutics, Rutgers Robert Wood Johnson Medical School, Piscataway, NJ 08854; m.mouradian@rutgers.edu disney@scripps.edu.ORCID 0000-0002-9937-412X
Matthew D DisneyDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458; m.mouradian@rutgers.edu disney@scripps.edu.ORCID 0000-0001-8486-1796
Scripps Research Institute · USRutgers, The State University of New Jersey · USIowa State University · US
Funding
Using a disease-affected cell to synthesize its own drugDP1NS096898 · NINDS · SCRIPPS FLORIDA · PI DISNEY, MATTHEW D · 2015 to 2019
$5.0MSequence-based Design of Small Molecules Targeting RNAR01GM097455 · NIGMS · SCRIPPS FLORIDA · PI DISNEY, MATTHEW D · 2012 to 2019
$4.6MFinding, folding and characterizing the functions of disease-relevant RNA structuresR01GM133810 · NIGMS · IOWA STATE UNIVERSITY · PI Walter Moss · 2019 to 2026
$2.8MManipulating Gene Expression in the Dyskinesias of Parkinson's DiseaseR01NS073994 · NINDS · EMORY UNIVERSITY · PI MOURADIAN, M. MARAL, PAPA, STELLA M · 2011 to 2016
$2.4MPP2A Dysregulation in the Pathogenesis of alpha-SynucleinopathiesR01NS101134 · NINDS · RBHS-ROBERT WOOD JOHNSON MEDICAL SCHOOL · PI MOURADIAN, M. MARAL, NICHOLLS, RUSSELL E · 2017 to 2021
$2.3MSynergistic Neuroprotective Mechanisms of Coffee Components in Parkinson's DiseasR01AT006868 · NCCIH · UNIV OF MED/DENT NJ-R W JOHNSON MED SCH · PI MOURADIAN, M. MARAL, STOCK, JEFFRY B · 2012 to 2016
$2.2MAlpha-Synuclein Regulation by microRNAsR01NS070898 · NINDS · UNIV OF MED/DENT NJ-R W JOHNSON MED SCH · PI JUNN, EUNSUNG · 2011 to 2015
$1.7MRNA Targeted Drug Discovery and Development for Parkinson DiseaseUH3NS116921 · NINDS · RBHS-ROBERT WOOD JOHNSON MEDICAL SCHOOL · PI DISNEY, MATTHEW D, MOURADIAN, M. MARAL · 2021 to 2021
$851kDeveloping Novel RNA-Based Therapeutics for Parkinson's DiseaseR33NS096032 · NINDS · RBHS-ROBERT WOOD JOHNSON MEDICAL SCHOOL · PI DISNEY, MATTHEW D, MOURADIAN, M. MARAL · 2018 to 2019
$843kUnderstanding Epstein Barr virus oncogenicity through non-coding RNA structureR00GM112877 · NIGMS · IOWA STATE UNIVERSITY · PI MOSS, WALTER · 2017 to 2019
$747kDeveloping Novel RNA-Based Therapeutics for Parkinson's DiseaseR21NS096032 · NINDS · RBHS-ROBERT WOOD JOHNSON MEDICAL SCHOOL · PI DISNEY, MATTHEW D, MOURADIAN, M. MARAL · 2017 to 2017
$452kTranslational regulation by DJ-1R21NS095003 · NINDS · RBHS-ROBERT WOOD JOHNSON MEDICAL SCHOOL · PI JUNN, EUNSUNG · 2016 to 2017
$437kNCCIH NIH HHS R01 AT006868NIGMS NIH HHS R00 GM112877NIGMS NIH HHS R01 GM097455NIGMS NIH HHS R01 GM133810NINDS NIH HHS DP1 NS096898NINDS NIH HHS R01 NS070898NINDS NIH HHS R01 NS073994NINDS NIH HHS R01 NS101134NINDS NIH HHS R21 NS095003NINDS NIH HHS R21 NS096032NINDS NIH HHS R33 NS096032NINDS NIH HHS UH3 NS116921
Abstract
Many proteins are refractory to targeting because they lack small-molecule binding pockets. An alternative to drugging these proteins directly is to target the messenger (m)RNA that encodes them, thereby reducing protein levels. We describe such an approach for the difficult-to-target protein α-synuclein encoded by the
Indexed as
Response Elements3' Untranslated Regionsalpha-SynucleinAnimalsCell Line, TumorHumansIntrinsically Disordered ProteinsMiceProtein BiosynthesisProtein Synthesis InhibitorsRNA, Messenger3' Untranslated Regionsalpha-SynucleinIntrinsically Disordered ProteinsProtein Synthesis InhibitorsRNA, Messengerchemical biologyintrinsically disordered proteinsParkinson’s diseaseRNAα-synuclein
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.