Antisense oligonucleotides: translation from mouse models to human neurodegenerative diseases
Schoch, Kathleen M., Miller, Timothy M.·2017·Neuron
Cited by (274)
Items 201–274 of 274
- Antisense oligonucleotides as drugs with both direct and indirect antiviral actionsJin Hong, Vivek K. Rajwanshi · 2025 · Antiviral Research
- CpG oligonucleotides induce acute murine thrombocytopenia dependent on toll-like receptor 9 and spleen tyrosine kinase pathwaysKarl E. Johansson, Amal Maouia, Johan Rebetz, Geneviève Marcoux, Oonagh Shannon, Joseph E. Italiano, Padma Narayanan, Scott P. Henry, Lijiang Shen, John W. Semple · 2024 · Journal of Thrombosis and Haemostasis
- Splicing Modulation as a Promising Therapeutic Strategy for Lysosomal Storage Disorders: The Mucopolysaccharidoses ExampleJuliana Inês Santos, Mariana Gonçalves, Liliana Matos, Luciana Moreira, Sofia Carvalho, Maria João Prata, Maria Francisca Coutinho, Sandra Alves · 2022 · Life
- The Role of Human Pluripotent Stem Cells in Amyotrophic Lateral Sclerosis: From Biological Mechanism to Practical ImplicationsLaura Ceccarelli, Lorenzo Verriello, Giada Pauletto, Mariarosaria Valente, Leopoldo Spadea, Carlo Salati, Marco Zeppieri, Tamara Ius · 2024 · Frontiers in Bioscience-Landmark
- Frontiers of Neurodegenerative Disease Treatment: Targeting Immune Cells in Brain Border RegionsSenthil Kumaran Satyanarayanan, Zixu Han, Jingwei Xiao, Qiuju Yuan, Wing‐Ho Yung, Ya Ke, Raymond Chuen‐Chung Chang, Maria Huachen Zhu, Huanxing Su, Kuan Pin Su et al. · 2024 · Brain Behavior and Immunity
- In Vivo Administration of Therapeutic Antisense OligonucleotidesLuisa Statello, Mohamad Moustafa Ali, Chandrasekhar Kanduri · 2020 · Methods in molecular biology
- Pyrrole–Imidazole Polyamides – A Frontrunner in Nucleic Acid‐Based Small Molecule DrugsThangavel Vaijayanthi, Ganesh N. Pandian, Hiroshi Sugiyama · 2023 · Advanced Therapeutics
- Antisense oligonucleotide-mediated terminal intron retention of endoglin: A potential strategy to inhibit renal interstitial fibrosisTessa Gerrits, Kyra L. Dijkstra, Jan Anthonie Bruijn, Marion Scharpfenecker, Roel Bijkerk, Hans J. Baelde · 2024 · Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease
- Lattice Corneal DystrophyMajid Moshirfar, William B. West, Yasmyne C. Ronquillo · 2021 · StatPearls
- Antisense Oligonucleotide Therapeutics for Neurodegenerative DisordersClaudia M. Testa · 2021 · Current Geriatrics Reports
- Antisense‐induced knockdown of cAMP response element‐binding protein downregulates Per1 gene expression in the shell region of nucleus accumbens resulting in reduced alcohol consumption in miceRishi Sharma, Vaibhav Kumar Mishra, Meet S Parikh, Anshul Soni, Pradeep Kumar Sahota, Mahesh M. Thakkar · 2021 · Alcoholism Clinical and Experimental Research
- Virus-like siRNA construct dynamically responsive to sequential microenvironments for potent RNA interferenceYue Wang, Xiujue Zheng, Jun Liu, Li Chen, Qixian Chen, Yan Zhao · 2022 · Journal of Colloid and Interface Science
- The Novel LncRNA WASH5P Inhibits Colorectal Cancer Carcinogenesis via Targeting AKT Signaling PathwayHongyun Wei, Tao Mao, Qian Zhang, Keyu Ren, Xingsi Qi, Yunmei Zhang, Bin Cao, Yanchun Jin, Zibin Tian, Linlin Ren · 2022 · Frontiers in Oncology
- Targeted Delivery of Nucleic Acid Therapeutics: Emerging Carriers and Applications in Common Metabolic and Inflammatory DiseasesXiyuan Lin, Li Chen, Keran Jia, Shengbiao Li, Qiuhong Li, Jingyan Yi, Jinshan Xing · 2026 · International Journal of Nanomedicine
- Antisense Oligonucleotides in Parkinson's Disease: Challenges, Opportunities, and the Promise of Low‐Intensity Focused Ultrasound for Brain DeliveryShunsuke Obika, Javier Blesa · 2026 · Journal of Neurochemistry
- Oligonucleotide-Recognizing Topoisomerase Inhibitors (OTIs): Precision Gene Editors for Neurodegenerative Diseases?B.D. Bax, Dmitry A. Sutormin, Neil Q. McDonald, Glenn A. Burley, Tatyana A. Shelkovnikova · 2022 · International Journal of Molecular Sciences
- CRISPR interference to evaluate modifiers of C9ORF72-mediated toxicity in FTDSarah R. Pickles, Desiree Zanetti Alepuz, Yuka Mitsuhashi Koike, Mei Yue, Jimei Tong, Pinghu Liu, Yugui Zhou, Karen R. Jansen‐West, Lillian M. Daughrity, Yuping Song et al. · 2023 · Frontiers in Cell and Developmental Biology
- A brief review of ALS from an immunological perspective with potential immunotherapy optionsGülsüm Ayhan, Esra Aydemir, Furkan Ayaz · 2025 · Discover Immunity.
- Enhancing the delivery of SOD1 antisense oligonucleotides to the murine brain using lipid nanoparticles and image-guided focused ultrasoundGayathri R. Ediriweera, Nicholas L. Fletcher, Gary Cowin, Joshua D. Simpson, Liyu Chen, Joanna Marta Wasielewska, Jeremy Stephen Lum, Luke McAlary, Mikayla L. Brown, Justin J. Yerbury et al. · 2022 · Research Square
- PROGRESS OF OLIGONUCLEOTIDE THERAPEUTICS TARGET TO RNAYufei Pan, Xinyang Zhou, Zhenjun Yang · 2022 · Wiley eBooks
- Terra incognita of glial cell dynamics in the etiology of leukodystrophies: Broadening disease and therapeutic perspectivesRishabh Chaudhary, Mujeeba Rehman, Vipul Agarwal, Anand Kumar, Arjun Singh Kaushik, Siddhi Srivastava, Sukriti Srivastava, Rajkumar Verma, Paruvathanahalli Siddalingam Rajinikanth, Vikas Mishra · 2024 · Life Sciences
- ClinASO: An open-source platform for rapid drug discovery of gapmer antisense oligonucleotidesShunkai Chen, Hao Liu, Dezi Cong, Ao Dong, Yuhang Wang, Jingyi Bi, Shijie Guo, Juan Yang, Xiaolei Wang, Guiping Ren et al. · 2026 · Molecular Therapy — Nucleic Acids
- Ext1-targeted gene therapy ameliorates tauopathy by inhibiting heparan sulfate-dependent tau propagationRongyao Huang, Xiaoxun Ye, Jiayi Liu, Chunyan Yang, Xiaoya Meng, Tingting Dou, Xiaoqing Zhuang, Jinghan Yang, Naizhen Zheng, Yang Zhao et al. · 2026 · Pharmacological Research
- Amyloidosis of the heart: pathophysiology, diagnosis, and treatmentAndy Yi-Yang Wang, Uzair Mahmoud, Jared M. Feldman, Stephen Pan, Wilbert S. Aronow, Diwakar Jain · 2025 · Expert Opinion on Pharmacotherapy
- Antisense Oligonucleotide-Mediated Downregulation of IGFBPs Enhances IGF-1 SignalingAlper Yavas, Maaike van Putten, Annemieke M Aartsma-Rus · 2024 · Journal of Neuromuscular Diseases
- Antisense Oligonucleotides in Psychiatric DisordersHeiko Graf, Claudia D. Wurster · 2018 · Journal of Clinical Psychopharmacology
- Genspezifische Therapien neurologischer Erkrankungen – aktueller Stand und PerspektivenDávid Brenner, Albert Christian Ludolph · 2020 · Der Nervenarzt
- Advances in the understanding of hereditary ataxia – implications for future patientsAnna Maria Zeitlberger, Heather Ging, Suran Nethisinghe, Paola Giunti · 2018 · Expert Opinion on Orphan Drugs
- Enclosure of siRNA in Alternately Hydrophilic–Hydrophobic Double-Layered Nanoarchitectures for Promoted RNAiJinxuan He, Zhixiang Mou, Yuchen Tian, Liangping Yu, Tianjun Guan, Qixian Chen, Lan Chen · 2022 · ACS Applied Polymer Materials
- How can MSI enhance our understanding of ASO distribution?Laura van der Vloet, Pierre Barbier Saint Hilaire, Christophe Bouillod, Emre Mehmet Isin, Ron M. A. Heeren, Michiel Vandenbosch · 2024 · Drug Discovery Today
- Therapeutic suppression of Tubb4a rescues H-ABC leukodystrophySunetra Sase, Julia Liao Hacker, Prabhat R. Napit, Anjali Bhagavatula, Sarah Woidill, Annemarie D’Alessandro, Marisa A. Jeffries, Akshata Almad, Asako Takanohashi, Quasar Saleem Padiath et al. · 2026 · Molecular Therapy
- Estimation of human brain fluid clearance using intrathecal imaging contrast agentsPer Kristian Eide, Aslan Lashkarivand, Susanne Sørensen Hernes, Geir André Ringstad, Markus Herberg Hovd · 2026 · Fluids and Barriers of the CNS
- Targeting neuroinflammation and neurodegeneration in Parkinson’s disease: Emerging natural and synthetic therapeutic strategiesSafia Arbab, Hanif Ullah, Yanting Han, Jiao Zhou, Ka Li · 2026 · Ageing Research Reviews
- ATF3/SOX11-regulated FST activates IGF1R–ERK/AKT–Sp1 signaling to sustain trigeminal neuropathic painYuejuan Ling, Bing Zhu, Zuo-hao Yao, Ling-Jie Ma, Lin-peng Zhu, Jing Zhang, Fei-fei Xu, Hao Wu, Lulu Ji, Yong‐Jing Gao · 2026 · Acta Pharmacologica Sinica
- α-Synuclein burden amplification in Parkinson’s disease: a unified genetic, molecular, and cellular frameworkSachiko Noda, Nobutaka Hattori · 2026 · Frontiers in Neurology
- MiR-129-5p: a novel therapeutic target for amyotrophic lateral sclerosis?Zachary C. E. Hawley, Danae Campos‐Melo, Michael Joseph Strong · 2020 · Non-coding RNA Investigation
- Antisense Oligonucleotide Therapy for Progressive Myoclonus EpilepsiesSilvia Nitschke, Berge Arakel Minassian · 2024
- Translocation of Oligonucleotide–Oligosaccharide Complexes into Cells of the BrainН. Н. Дыгало, Tatyana S. Kalinina, Dmitriy A. Lanshakov · 2018 · Doklady Biochemistry and Biophysics
- Antisense-Oligonukleotide IClaudia D. Wurster, Albert Christian Ludolph, Denise Lehmann, Heiko Graf · 2018 · Nervenheilkunde
- Investigation of Brain Functions Using Genetically Encoded ToolsН. Н. Дыгало · 2020 · Neuroscience and Behavioral Physiology
- Current Emerging Therapy for Amyloidosis NeuropathyYung‐Chih Cheng · 2020 · Neuro - Open Journal
- Antisense-Oligonukleotide IIDOROTHEE LEHMANN, AC Ludolph, Claudia D. Wurster, Heiko Graf · 2018 · Nervenheilkunde
- Elucidating the consequence and cause of microRNA dysregulation in amyotrophic lateral sclerosis (ALS)Zachary C. E. Hawley · 2020 · Scholarship@Western (Western University)
- Zgłębiając zawiłości struktur, funkcji i lekowalności cis motywów RNA u koronawirusówJoanna Sztuba-Solińska, Devadatta Gosavi · 2021 · Postępy Biochemii
- Medicinal Applications of Antisense Oligonucleotides: A ReviewKavya Pandiri, Mohammed Abdul Samad, Nadeem Abbas Gulamus, Hajera Khanam · 2020 · International Journal of Applied Pharmaceutical Sciences and Research
- Faculty Opinions recommendation of Emerging new concepts of degrader technologies.Peter R Bernstein · 2020 · Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
- Faculty Opinions recommendation of Oligonucleotide therapy mitigates disease in spinocerebellar ataxia type 3 mice.Alexandra Dürr · 2018 · Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
- Faculty Opinions recommendation of Targeting RNA: A transformative therapeutic strategy.Sue Fletcher · 2019 · Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
- Evaluating the efficacy of commercially available antisense oligonucleotides to reduce mouse and human tau in vivoPranav Vemula, Kathleen M. Schoch, Timothy M. Miller · 2022 · bioRxiv (Cold Spring Harbor Laboratory)
- Nucleic Acid Pharmaceutical AgentsRebecca A. Kohnken, Carole E. Harbison, Stéphanie Klein, Jeffery A. Engelhardt · 2023 · Elsevier eBooks
- ESCLEROSE LATERAL AMIOTRÓFICA (ELA): MECANISMOS, DIAGNÓSTICO E POSSÍVEIS TRATAMENTOSMaria Clara Oliveira Padilha Diniz, Adélcio Machado dos Santos, Barbara Gabrielly Galindo Guimarães Tribuzi Lula, Fernando Portes Peixoto, Francisco Luan Nogueira Alves, Gabriela Pereira Valadares, Ísis Cavalcante Amaral de Siqueira, Ivna Rocha Bastos, Lívia Maria Figueiredo Teles de Araújo, Lucas Galvão Carvalho et al. · 2023 · Revista Foco
- Preclinical Evaluation of Neuraxial Drugs for SafetyTony L. Yaksh, Robert B. Boyd, Orion P. Keifer · 2023 · Springer eBooks
- Klinik NörolojiMehmet Türkay Karcı, Erdem Özyurt, Merve Bahar Ercan, Safiye Gül Kenar, Filiz Demirdöğen, Betül Özenç, Özlem Ergin Beton, Nur Türkmen, Mustafa Şen, Mustafa Açıkgöz et al. · 2022 · Akademisyen Kitabevi eBooks
- Therapeutic strategies based on genetic interventionsRena C. Baek, Patrick F. Finn, Paolo G.V. Martini · 2024 · Elsevier eBooks
- Artemia Cyst Production in Solar Earthen Ponds in Vinh Chau, Vietnam, and Its Associated MicrobiomeTung Hoang, Binh Thai Nguyen, Thu Thi Minh Vo, Trung Tien Le, Long Minh Tran · 2025 · Aquaculture Research
- Insights From a Scoping Review and Bibliometric Analysis of Trends and Advances in Biomarker Research: The Role of MicroRNAs in Cognitive Decline Across Neurodegenerative DiseasesSamuel Reyes-Long, Ernesto Roldán-Valadéz, José Luis Cortes-Altamirano, Denise Clavijo‐Cornejo, Alfonso Alfaro-Rodríguez · 2025 · Cureus
- Antisense oligonucleotide of cargo adaptor BICD2 long-term effectively alleviates neuropathic pain via activation of PSD95 in the mouse spinal dorsal hornZixuan Wu, Yang Xu, Jing Xie, Gan Ma, Yu-Xia Li, Tong Yang, Jia-Hui Liang, Yang Li, Qihui Wang, Ling-Yun Hao et al. · 2025 · Neuropharmacology
- Additional file 5 of CRISPRi-based radiation modifier screen identifies long non-coding RNA therapeutic targets in gliomaLiu, S. John, Martina Malatesta, Brian V. Lien, Parna Saha, Shivani S. Thombare, Hong, Sung Jun, Leslie Pedraza, Koontz, Mark, Kyounghee Seo, Max A. Horlbeck et al. · 2020 · Figshare
- Additional file 1 of CRISPRi-based radiation modifier screen identifies long non-coding RNA therapeutic targets in gliomaLiu, S. John, Martina Malatesta, Brian V. Lien, Parna Saha, Shivani S. Thombare, Hong, Sung Jun, Leslie Pedraza, Mark Koontz, Kyounghee Seo, Max A. Horlbeck et al. · 2020 · Figshare
- Antisense Molecules: Design, Chemistry, and Therapeutic InnovationsPrema Vediappan, Swetha Aravamudhan, Jocelyn Olivia Prabhakar David, Abitha Sri Ganapathy, Suryalakshmi Guhanathan · 2026 · Mini-Reviews in Medicinal Chemistry
- Gene Targeted Therapies for Neurodegenerative Disorders: Strategies and Implications in ALS and SMAAyşe Yeşbek Kaymaz, Gamze Bora, Hayat Erdem‐Yurter, Christopher Grunseich · 2026 · Genes
- Thérapie génique pour l’amyotrophie spinale à l’aide de vecteur AAVAurore Besse · 2019
- Le développement préclinique des tcDNA pour la Dystrophie Musculaire de DuchennePhilippine Aupy · 2019
- Targeting ZNF703 in Luminal BBreast cancer by Antisense OligonucleotidesSandra Oluchi Udu-Ituma · 2023
- Protein kinase C in neurodegenerative disordersAzadeh Hekmat, Ali Akbar Saboury · 2026 · Elsevier eBooks
- The Enigma of Big Tau Exon 4a: Genomic Architecture, Biophysical Identity, and Unique Evolutionary MechanismsItzhak Fischer · 2026 · Biomolecules
- Immune cells and nanotechnology: Innovations in therapyRuth Naomi, Santhra Segaran Balan, Saiful Irwan Zubairi, Shariff Halim, Pamela Rosie David, Roslee Rajikan · 2026 · Elsevier eBooks
- Experimental epilepsy models for mechanistic investigation and translational researchAli Osman Arslan, Sevdenur Uzun, Güven Akçay · 2026 · Discover Neuroscience
- Recent Innovations in Drug Delivery Systems for Amyotrophic Lateral Sclerosis: Past, Present and FutureDhvani Padhiyar, Shital Butani, Tushar Vyas, Amit Mukharya, Amarjitsing Rajput · 2026 · AAPS PharmSciTech
- Development of Antisense Oligonucleotide Gapmers for the Treatment of Dyslipidemia and LipodystrophyTejal Aslesh, Toshifumi Yokota · 2026 · Methods in molecular biology
- Olezarsen for the Treatment of HypertriglyceridemiaAntonio Sebastian Hernandez Rodriguez, Toshifumi Yokota · 2026 · Methods in molecular biology
- Development of Antisense Gapmers for the Treatment of Huntington’s DiseaseTejal Aslesh, Toshifumi Yokota · 2026 · Methods in molecular biology
- Biophysics-informed computational framework for predicting antisense oligonucleotide knockdown efficacy under small-data constraintsRisha Guha, Shawn Kim, Anwita Bandaru, Nithika Vivek · 2026 · Frontiers in Systems Biology
- New Treatments for Amyloidosis NeuropathyYung‐Chih Cheng · 2025 · Neuro - Open Journal