Cell-Selective Adeno-Associated Virus-Mediated SCN1A Gene Regulation Therapy Rescues Mortality and Seizure Phenotypes in a Dravet Syndrome Mouse Model and Is Well Tolerated in Nonhuman Primates

Tanenhaus, Annie, Stowe, Timothy, Young, Andrew, McLaughlin, John, Aeran, Rangoli, Lin, I. Winnie, Li, Jianmin, Hosur, Raghavendra +9 more·2022·Human Gene Therapy
DOI 10.1089/hum.2022.037

Cited by (124)

Items 101–124 of 124

  1. Gene therapy targets interneurons to tackle Dravet syndrome
    Angie Voyles Askham · 2022 · The Transmitter
  2. A hyperthermic seizure unleashes a surge of spreading depolarizations in Scn1a deficient mice
    Isamu Aiba, Yao Yu Ning, Jeffrey L. Noebels · 2022 · bioRxiv (Cold Spring Harbor Laboratory)
  3. The Future of SCN1A Gene-targeting Research for the Treatment of Dravet Syndrome
    Rajvinder Karda · 2023 · touchREVIEWS in Neurology
  4. Potentiating Na V 1.1 in Dravet syndrome patient iPSC-derived GABAergic neurons increases neuronal firing frequency and decreases network synchrony
    Matt R. Kelley, Laura B. Chipman, Shoh M. Asano, Matthew Knott, Samantha Howard, Allison P. Berg · 2023 · bioRxiv (Cold Spring Harbor Laboratory)
  5. Genes Encoding Ion Channels in Neurotherapeutics: Opportunities and Challenges
    Enes Akyüz, Habiba Eyvazova · 2023 · Springer eBooks
  6. AAV9-mediated targeting of natural antisense transcript as a novel treatment for Dravet syndrome
    Juan F. Antinao Diaz, Ellie M. Chilcott, Amanda Almacellas‐Barbanoj, Anna Keegan, Sonam Gurung, Valda Pauzuolyte, Zak Waddington, Maria Kyriacou, Amy McTague, Judith Helen Cross et al. · 2026 · Molecular Therapy — Nucleic Acids
  7. Using genetically diverse Collaborative Cross mouse strains to model SCN1A-related Dravet Syndrome
    Zaynab Shakkour · 2024
  8. SCN2A-Related Disorders
    Megan Abbott, Kevin J. Bender, Andreas Brunklaus, Scott Demarest, Shawn M. Egan, Isabel Haviland, Jennifer A. Kearney, Leah Schust Myers, Heather E. Olson, Stephan Sanders et al. · 2024 · Cambridge University Press eBooks
  9. Prenatal sodium channel dysfunction in Dravet syndrome alters cortical development
    Clara Zourray, James Mills, James Dominic Mills, Martina Mainardi, James Street, Joanna Bartkiewicz, Simeon R. Mihaylov, Jacopo Sartorelli, Nathanael O’Neill, Jenna Carpenter et al. · 2025 · Research Square
  10. Molecular genetics of benign adult familial myoclonus epilepsy
    Hiroyuki Ishiura · 2025 · Rinsho Shinkeigaku
  11. Two Halves Make a Whole SCN1A
    Jennifer C. Wong · 2025 · Epiliepsy currents/Epilepsy currents
  12. Modulating splicing in 5’ untranslated regions to treat rare haploinsufficient disease
    Eloise S. Beer Wells, Laura De Conti, Hyung Chul Kim, Narjes Rohani, V. Kartik Chundru, Laura M. Watts, Ruebena Dawes, Yuyang Chen, Alexandra C Martin-Geary, Michael J. Griffiths et al. · 2025 · bioRxiv (Cold Spring Harbor Laboratory)
  13. Advances of Genomic Medicine in Neurodevelopmental Disorders
    Hosneara Akter, Mohammed J. Uddin, A.H.M. Nurun Nabi · 2026 · Interdisciplinary biotechnological advances
  14. Revolutionizing Gene Therapy: An Explicative Review on Engineering Adeno-associated Virus Vectors
    Shankhadip Nandi, Shaibal Chandra, Kaushik Biswas, Kamalika Mazumder, Swarupananda Mukherjee, Dipanjan Karati, Soumendra Nath Bandyopadhyay, Pasquale De Toro · 2026 · Regenerative Engineering and Translational Medicine
  15. Exploring gene therapy for developmental and epileptic encephalopathies (DEEs): possibilities or promises?
    Anna Behr, Miguel Chillón · 2026 · Regenerative Medicine
  16. At the forefront of gene-based therapies in Dravet syndrome
    Antonella Riva, Sara Di Gioacchino, Greta Volpedo, Beatrice Risso, Antonella De Lillo, Federico Zara, Antonio Falace, Pasquale Striano · 2026 · Expert Opinion on Emerging Drugs
  17. News from Biotech
    2026 · Molecular Therapy
  18. AAV Vectors in Regenerative Medicine and Cellular Reprogramming: Potential, Pitfalls, and Specificity Constraints
    Mariam abdelnaby, Adelya A. Galiakberova, Erdem Bairovich Dashinimaev · 2026 · International Journal of Molecular Sciences
  19. ANTISENSE OLIGONUCLEOTIDE THERAPY FOR DRAVET SYNDROME: A CRITICAL REVIEW OF THE EVIDENCE FOR DISEASE MODIFICATION WITH ZOREVUNERSEN
    Oliwia Żmuda, Anna Halot, Patrycja Walo, Natalia Skórka, Magdalena Tatara, Karolina Skórka, Klaudia Wydra, Jakub Jakubiec, Michał Szubartowski, Adam Szymczyk · 2026 · International Journal of Innovative Technologies in Social Science
  20. Adeno‐associated virus ‐mediated restoration of Na V 1.1 in targeted brain regions ameliorates epilepsy in symptomatic Dravet syndrome mice
    Martina Mainardi, Simone Brusco, Serena Gea Giannelli, Vania Broccoli, Gaia Colasante · 2026 · Epilepsia
  21. Precision Therapeutics for Monogenic Epilepsies: From Molecular Mechanisms to Disease Modification in Developmental and Epileptic Encephalopathies and Neurodevelopmental Disorders with Epilepsy
    Michael Scott Perry · 2026 · Journal of Clinical Medicine
  22. Dravet Syndrome Updates: Evolving Insights and Emerging Therapeutics
    Stephanie Fukui, Kelly G. Knupp · 2026 · Current Neurology and Neuroscience Reports
  23. Molecular Mechanisms and Therapeutic Advances in SCN1A-Related Dravet Syndrome
    Ninger Zhang · 2026 · Advances in Clinical Medicine
  24. An engineered, compact Cas12m variant for multiplexed gene activation in vivo
    Hainan Zhang, Lin Yang, Yuling Liu, Dingyi Han, Yingnan Li, Jiajia Lin, Bingyan Wang, Jiaxu Hu, Yaru Zhang, Yi Zhang et al. · 2026 · Nature Biotechnology