ArticleProceedings of the National Academy of Sciences of the United States of America2024
Decreasing the intrinsically disordered protein α-synuclein levels by targeting its structured mRNA with a ribonuclease-targeting chimera.
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Article in Proceedings of the National Academy of Sciences of the United States of America, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers.
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31citing papers in PubMed
9.4field-weighted citation impact, top 1% of its field
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Who cites it
31 citing papers in PubMed, 40 citations in OpenAlex.
- Organoids: Key advances, optimization, and technological iterations in their application to neurodegenerative diseases.Neural regeneration research · 2026Article
- From synaptic development to degeneration: a narrative review of small molecule strategies targeting alpha-synuclein in Parkinson's disease.Metabolic brain disease · 2026Review
- Small-Molecule Targeting of the Iron-Responsive Element in the APP mRNA 5'-UTR to Control Amyloid Translation in Alzheimer's Disease.International journal of molecular sciences · 2026Review
- The application of drug delivery systems based on targeted protein degradation chimeras in disease treatment strategies.Journal of nanobiotechnology · 2026Review
- An orthogonal RNA aptamer for targeted RNA regulation in phase-separated subcellular compartments.Nature communications · 2026Article
- Targeted RNA Degradation by RIBOTACs: A Novel Therapeutic Avenue for Ophthalmic Diseases.International journal of molecular sciences · 2026Review
- Deciphering the regulatory landscape of enhancer RNAs in health and disease.Signal transduction and targeted therapy · 2026Review
- Predicting small molecule-RNA interactions without RNA tertiary structures.Nature biotechnology · 2026Article
- RNA Splicing as a Therapeutic Target in Cancer.Annual review of pharmacology and toxicology · 2026Review
- Programmable targeted RNA degradation via dCas13d-directed chaperone-mediated autophagy (dCasCMA).Nature communications · 2025Article
- SHAPE-Based Chemical Probes for Studying preQACS chemical biology · 2025Article
- Targeted RNA Degradation as a Promising Therapeutic Strategy.International journal of molecular sciences · 2025Review
- Targeted α-Synuclein mRNA Degradation by PMO-Based RNA-Degrading Chimeras.bioRxiv : the preprint server for biology · 2025Article
- Discovery of Macrocyclic Peptide Binders, Covalent Modifiers, and Degraders of a Structured RNA by mRNA Display.Journal of the American Chemical Society · 2025Article
- Targeting neurodegenerative disease-associated protein aggregation with proximity-inducing modalities.Acta pharmacologica Sinica · 2025Review
- Review
- Targeting the SARS-CoV-2 RNA Translation Initiation Element SL1 by Molecules of Low Molecular Weight.Journal of the American Chemical Society · 2025Article
- [Targeting chimera technology: A new tool for undruggable in breast cancer].Zhong nan da xue xue bao. Yi xue ban = Journal of Central South University. Medical sciences · 2025Review
- The Unified Theory of Neurodegeneration Pathogenesis Based on Axon Deamidation.International journal of molecular sciences · 2025Review
- The interplay of iron, oxidative stress, and α-synuclein in Parkinson's disease progression.Molecular medicine (Cambridge, Mass.) · 2025Review
Corrections and comments
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Authors and funding
15 authors at 2 institutions in 1 country.
Yuquan Tong *Department of Chemistry, The Scripps Research Institute, Jupiter, FL 33458.
Peiyuan Zhang *Department of Chemistry, The Scripps Research Institute, Jupiter, FL 33458.
Xueyi YangDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458.
Xiaohui LiuDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458.
Jie ZhangRutgers Robert Wood Johnson Medical School Institute for Neurological Therapeutics, Piscataway, NJ 08854.
Magda GrudniewskaRutgers Robert Wood Johnson Medical School Institute for Neurological Therapeutics, Piscataway, NJ 08854.ORCID 0000-0002-3971-8437
Ikrak JungRutgers Robert Wood Johnson Medical School Institute for Neurological Therapeutics, Piscataway, NJ 08854.
Daniel AbeggDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458.
Jun LiuRutgers Robert Wood Johnson Medical School Institute for Neurological Therapeutics, Piscataway, NJ 08854.
Jessica L Childs-DisneyDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458.
Quentin M R GibautDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458.
Hafeez S HaniffDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458.
Alexander AdibekianDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458.ORCID 0000-0001-6453-0244
M Maral MouradianRutgers Robert Wood Johnson Medical School Institute for Neurological Therapeutics, Piscataway, NJ 08854.ORCID 0000-0002-9937-412X
Matthew D DisneyDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458.ORCID 0000-0001-8486-1796
Scripps Research Institute · USRutgers, The State University of New Jersey · US
Funding
Design of Precision Small Molecules Targeting RNA Repeating Transcripts to Manipulate and Study Disease BiologyR35NS116846 · NINDS · UNIVERSITY OF FLORIDA · PI Matthew D Disney · 2020 to 2026
$9.4MUsing 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
$4.6MPP2A Dysregulation in the Pathogenesis of alpha-SynucleinopathiesR01NS101134 · NINDS · RBHS-ROBERT WOOD JOHNSON MEDICAL SCHOOL · PI MOURADIAN, M. MARAL, NICHOLLS, RUSSELL E · 2017 to 2021
$2.3MPME-1: Pathogenetic Role and Therapeutic Opportunity in Neurodegenerative Mixed ProteinopathiesRF1NS130702 · NINDS · RUTGERS BIOMEDICAL AND HEALTH SCIENCES · PI MOURADIAN, M. MARAL · 2023 to 2023
$1.7MProbing conformational changes by protein surface azidationR01GM145886 · NIGMS · UNIVERSITY OF ILLINOIS AT CHICAGO · PI ADIBEKIAN, ALEXANDER · 2022 to 2025
$1.6MRNA 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
$843kRNA Targeted Drug Discovery and Development for Parkinson DiseaseUG3NS116921 · NINDS · RBHS-ROBERT WOOD JOHNSON MEDICAL SCHOOL · PI DISNEY, MATTHEW D, MOURADIAN, M. MARAL · 2020 to 2020
$523kDeveloping 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
$452kRole of Transglutaminase 2 in SynucleinopathiesR21NS123770 · NINDS · RBHS-ROBERT WOOD JOHNSON MEDICAL SCHOOL · PI MOURADIAN, M. MARAL · 2021 to 2021
$432kAlzheimer’s Disease Protection by Reduced Adenylyl Cyclase Type 5R21AG075656 · NIA · RUTGERS BIOMEDICAL AND HEALTH SCIENCES · PI MOURADIAN, M. MARAL, VATNER, STEPHEN F · 2022 to 2022
$432kNIA NIH HHS R21 AG075656NIDA NIH HHS R03 DA033977NIGMS NIH HHS R01 GM097455NIGMS NIH HHS R01 GM145886NINDS NIH HHS DP1 NS096898NINDS NIH HHS R01 NS101134NINDS NIH HHS R21 NS096032NINDS NIH HHS R21 NS123770NINDS NIH HHS R33 NS096032NINDS NIH HHS R35 NS116846NINDS NIH HHS RF1 NS130702NINDS NIH HHS UG3 NS116921NINDS NIH HHS UH3 NS116921
Abstract
α-Synuclein is an important drug target for the treatment of Parkinson's disease (PD), but it is an intrinsically disordered protein lacking typical small-molecule binding pockets. In contrast, the encoding
Indexed as
Intrinsically Disordered ProteinsParkinson Disease5' Untranslated Regionsalpha-SynucleinHumansRibonucleasesRNA, Messenger5' Untranslated Regionsalpha-SynucleinIntrinsically Disordered ProteinsRibonucleasesRNA, MessengerAlzheimer’sparkinsonismRNAtargeted degradationα-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.