Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models

Cited by (279)

Items 201–279 of 279

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  5. Theme 7 Pre-clinical therapeutic strategies
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  7. Enhancing the delivery of SOD1 antisense oligonucleotides to the murine brain using lipid nanoparticles and image-guided focused ultrasound
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  9. MOLECULAR DOCKING STUDIES OF MONOMERIC WILDTYPE AND MUTANT (H81A, H49R) SOD1 WITH EDARAVONE AND RILUZOLE
    RADHIKA NAGRE, VAEESHNAVI BUWA · 2022 · International Journal of Chemistry Research
  10. Tofersen: Silver Lining or Hyperbole??
    Tanushree Chawla, Vinay Goyal · 2023 · Annals of Indian Academy of Neurology
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  12. A NOTCH3 pathogenic variant influences osteogenesis and can be targeted by antisense oligonucleotides in induced pluripotent stem cells
    ERNESTO M. CANALIS, Jungeun Yu, Lauren Schilling, Paymaan Jafar‐Nejad, Michele Carrer · 2025 · PLoS ONE
  13. SNP Analysis of the SOD1 gene involved in Amyotrophic Lateral Sclerosis (ALS)
    Hetal Patel, Anandita Banerjee, Pritha Saha, Subramaniyan Divya, Palaniyandi Ravanan, Priti Talwar · 2025 · Journal of Taibah University for Science
  14. Tofersen - A Ray of Hope for ALS Patients : A Mini Review
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  15. Local administration of a novel siRNA modality into the CNS extends survival and improves motor function in the SOD1 G93A mouse model for ALS
    Chunling Duan, Moorim Kang, Xiaojie Pan, Zubao Gan, Vera Huang, Guanlin Li, Robert F. Place, Long‐Cheng Li · 2023 · bioRxiv (Cold Spring Harbor Laboratory)
  16. Enclosure of siRNA in Alternately Hydrophilic–Hydrophobic Double-Layered Nanoarchitectures for Promoted RNAi
    Jinxuan He, Zhixiang Mou, Yuchen Tian, Liangping Yu, Tianjun Guan, Qixian Chen, Lan Chen · 2022 · ACS Applied Polymer Materials
  17. Cell and gene therapy for amyotrophic lateral sclerosis
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  18. miR-146a is a pleiotropic regulator of motor neuron degeneration
    Dylan A. Galloway, Hunter L. Patterson, Mariah L. Hoye, Tao Shen, Mark Shabsovich, Kathleen M. Schoch, Cindy V. Ly, Jonathan S. Mill · 2026 · Proceedings of the National Academy of Sciences
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  22. Intravenous administration of an engineered AAV9-gene-silencing vector suppresses human SOD1 and extends survival in an ALS mouse model
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  24. Neurotoxicity and ALS: Insights into Pathogenesis
    Steve Vucic, Matthew C. Kiernan · 2021 · Springer eBooks
  25. Antisense oligonucleotide drugs for neurological and neuromuscular disease
    Roger M. Lane, C. Frank Bennett · 2020 · Elsevier eBooks
  26. Molecular and Genetic Therapies
    Pushpa Narayanaswami, Saša Živković · 2021 · Elsevier eBooks
  27. Amyotrophic Lateral Sclerosis: Neurochemical Biomarkers
    Federico Verde, Nicola Ticozzi · 2022 · Springer eBooks
  28. Antisense Oligonucleotides: Concepts and Pharmaceutical Applications
    A Kenneth Araya, David Arias, Karla Coto, María Rebeca López, Joselyn Rivera, Juan José Mora · 2023 · Borneo Journal of Pharmacy
  29. Antisense oligonucleotides targeting a NOTCH3 mutation in male mice ameliorate the cortical osteopenia of lateral meningocele syndrome
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  30. Superoxide Dismutase and Catalase
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  31. Cure the Incurable: Update of Treatment in Inherited Neuromuscular Disorders
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  32. Maximizing the translational potential of neurophysiology in amyotrophic lateral sclerosis: a study on compound muscle action potentials
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  35. Neuroprotection in Amyotrophic Lateral Sclerosis
    Kewal K. Jain · 2019 · Springer protocols handbooks/Springer protocols
  36. Neurotoxicity and ALS: Insights into Pathogenesis
    Steve Vucic, Matthew C. Kiernan · 2022 · Springer eBooks
  37. Expression of a miRNA targeting mutated SOD1 in astrocytes induces motoneuron plasticity and improves neuromuscular function in ALS mice
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  38. A Cultured Sensorimotor Organoid Model Forms Human Neuromuscular Junctions
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  39. HuD (ELAVL4) gain-of-function impairs neuromuscular junctions and induces apoptosis in in vitro and in vivo models of amyotrophic lateral sclerosis
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  40. Antisense treatment alleviates ALS in animals
    Ian Fyfe · 2018 · Nature Reviews Neurology
  41. Introduction to Novel Motor Neuron Disease
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  42. Development of a molecular therapy for the SOD1 familial variant of ALS
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  43. A copper chaperone-mimetic polytherapy for SOD1-associated amyotrophic lateral sclerosis
    Luke McAlary, Victoria K. Shephard, Gareth S. A. Wright, Justin J. Yerbury · 2021 · bioRxiv (Cold Spring Harbor Laboratory)
  44. Theme 07 - PRE-CLINICAL THERAPEUTIC STRATEGIES
    2021 · Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
  45. Faculty Opinions recommendation of Antisense oligonucleotide therapies for neurodegenerative diseases.
    Matthew David Disney · 2019 · Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
  46. Faculty Opinions recommendation of Scn8a Antisense Oligonucleotide Is Protective in Mouse Models of SCN8A Encephalopathy and Dravet Syndrome.
    Laurent Villard · 2020 · Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
  47. IV. Gene Therapy for Amyotrophic Lateral Sclerosis
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  48. Therapeutic strategies based on genetic interventions
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  49. Maximising the translational potential of neurophysiology in amyotrophic lateral sclerosis: a study on compound muscle action potentials
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  50. Antisense oligonucleotide drugs for neurological and neuromuscular diseases
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  52. SCAD: A Modular Platform for Efficient Delivery of Duplex RNA to the CNS and Beyond
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  53. Performing Compound Motor Action Potential Measurement in Zebrafish: A Description of Methodology and a Comparison Between Healthy and ALS ‐Affected Animals
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  54. BLOC1S1 depletion via splice-switching oligonucleotides improves mitochondrial respiration and rescues ALS phenotypes
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  55. Additional file 1 of Neurofilaments in motor neuron disorders: towards promising diagnostic and prognostic biomarkers
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  58. Gene-targeted versus broad-spectrum therapies in ALS: comparative lessons and strategic outlook
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  59. Lumbar Intrathecal Injection of SOD1-ASOs for Precise CNS Targeting and Predictive Efficacy in Human SOD1-G93A ALS Mice
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