Durable suppression of seizures in a preclinical model of <scp>KCNT1</scp> genetic epilepsy with divalent <scp>small interfering RNA</scp>
Patients with KCNT1-driven epilepsy experience up to hundreds of seizures per day and have severe impairment in cognitive, motor, and language development and high mortality. The dose-dependent efficacy and long durability of ATL-201 in mice show promise for ATL-201 as a disease-modifying treatment of KCNT1 epilepsy.
Authors
- Christopher Dejesus (ORCID: https://orcid.org/0000-0002-0678-6601)
- Garth A. Kinberger
- Jennifer S Lin (ORCID: https://orcid.org/0000-0001-9169-7410)
- Matthew L. Rook (ORCID: https://orcid.org/0000-0002-5332-7678)
- Aimee L. Jackson (ORCID: https://orcid.org/0000-0002-3786-7391)
- Mariam Traoré (ORCID: https://orcid.org/0009-0006-7900-4793)
- Benjamin J. Andreone
- Bryce Howat
- Corrie L. Gallant‐Behm (ORCID: https://orcid.org/0000-0003-0915-2847)
- Thomas W. Lynch
- Phensinee Haruehanroengra
- Jenna Tocci
- Hryhoriy Zhoba
- Kelly A. Rogers
- Matthew Deninger
- Guillermo A. Yudowski (ORCID: https://orcid.org/0000-0001-5989-4131)
- Amr Omer (ORCID: https://orcid.org/0000-0001-7123-0274)
- Chunhua Yang (ORCID: https://orcid.org/0000-0003-0805-1008)
- Mingwei Li (ORCID: https://orcid.org/0000-0001-5851-6091)
- Qingmin Chen (ORCID: https://orcid.org/0000-0002-5645-3338)
- Stefan McDonough
- Alex Prinzen
- Gregory Miglis
- Lauren M. Carito
Publication Details
- Journal
- Epilepsia
- Published
- 2025-01-27
- DOI
- https://doi.org/10.1111/epi.18278
- Citations
- 4
- Primary Topic
- Advanced biosensing and bioanalysis techniques
- Type
- article
- Field-Weighted Citation Impact
- 3.22