Antisense oligonucleotide-mediated knockdown therapy in two infants with severe KCNT1 epileptic encephalopathy.

1. Division of Genetics and Genomics, Boston Children’s Hospital, Boston, MA USA
2. Nucleotide and Peptide Drug Discovery Center, Institute of Science Tokyo, Tokyo, Japan
3. Department of Neurology, Boston Children’s Hospital, Boston, MA USA
4. The Florey Institute of Neuroscience and Mental Health, University of Melbourne, Melbourne, Victoria Australia
5. Comprehensive Epilepsy Center, Department of Neurology, Yale School of Medicine, New Haven, CT USA
6. Department of Pharmacology, Yale School of Medicine, New Haven, CT USA
7. Department of Pharmacology, Feinberg School of Medicine, Northwestern University, Chicago, IL USA
8. Clinical Research Operations Center, Boston Children’s Hospital, Boston, MA USA
9. Department of Anesthesia, Critical Care, and Pain Medicine, Boston Children’s Hospital, Boston, MA USA
10. Department of Radiology, Boston Children’s Hospital, Boston, MA USA
11. Manton Center for Orphan Disease, Boston Children’s Hospital, Boston, MA USA
12. Harvard Medical School, Boston, MA USA
13. Broad Institute of MIT and Harvard, Cambridge, MA USA
14. R&D Servier Institute Paris-Saclay, Saclay, France
15. Department of Neurosurgery, Boston Children’s Hospital, Boston, MA USA
16. Department of Cellular and Molecular Physiology, Yale School of Medicine, New Haven, CT USA

@article{nakayama2026antisense,
author = {Nakayama, Tojo and El Achkar, Christelle M and Burbano, Lisseth E and Quraishi, Imran H and Wu, Jing and Li, Melody and Asami, Yutaro and Golinski, Sean R and Sherrill, Emma and Goodlett, Benjamin D and de Gusmao, Claudio M and Friedman, Danielle A and Lentucci, Claudia and Suslovitch, Victoria and Riccardi, Olivia and Faour, Kamli N W and Kuniholm, Ashley and Soucy Verran, Aubrie and Coffman, Siobhan and Ahtam, Banu and Zhao, Boxun and Chin, Diana H and DiDonato, Renata L and Hu, Chunguang A and Lopez, Edith and Hills, Sonia and Maljevic, Snezana and Tran, Helene and Bush, Lynn W and Grant, Patricia Ellen and Madsen, Joseph R and Smith, Richard S and Kaczmarek, Leonard K and Berde, Charles B and Yu, Timothy W},
title = {{Antisense oligonucleotide-mediated knockdown therapy in two infants with severe KCNT1 epileptic encephalopathy}},
journal = {Nature medicine},
year = {2026},
month = apr,
volume = {32},
number = {4},
pages = {1411--1420},
publisher = {Nature Portfolio},
issn = {1078-8956},
doi = {10.1038/s41591-026-04314-9},
url = {https://doi.org/10.1038/s41591-026-04314-9},
pmid = {41981306},
pmcid = {PMC13099374}
}

TY - JOUR
AU - Nakayama, Tojo
AU - El Achkar, Christelle M
AU - Burbano, Lisseth E
AU - Quraishi, Imran H
AU - Wu, Jing
AU - Li, Melody
AU - Asami, Yutaro
AU - Golinski, Sean R
AU - Sherrill, Emma
AU - Goodlett, Benjamin D
AU - de Gusmao, Claudio M
AU - Friedman, Danielle A
AU - Lentucci, Claudia
AU - Suslovitch, Victoria
AU - Riccardi, Olivia
AU - Faour, Kamli N W
AU - Kuniholm, Ashley
AU - Soucy Verran, Aubrie
AU - Coffman, Siobhan
AU - Ahtam, Banu
AU - Zhao, Boxun
AU - Chin, Diana H
AU - DiDonato, Renata L
AU - Hu, Chunguang A
AU - Lopez, Edith
AU - Hills, Sonia
AU - Maljevic, Snezana
AU - Tran, Helene
AU - Bush, Lynn W
AU - Grant, Patricia Ellen
AU - Madsen, Joseph R
AU - Smith, Richard S
AU - Kaczmarek, Leonard K
AU - Berde, Charles B
AU - Yu, Timothy W
TI - Antisense oligonucleotide-mediated knockdown therapy in two infants with severe KCNT1 epileptic encephalopathy
T2 - Nature medicine
J2 - Nat Med
PY - 2026
DA - 2026/04/14
VL - 32
IS - 4
SP - 1411
EP - 1420
SN - 1078-8956
PB - Nature Portfolio
DO - 10.1038/s41591-026-04314-9
UR - https://doi.org/10.1038/s41591-026-04314-9
LA - en
ER -

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