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Peer-reviewedLicence not statedScience Translational Medicine

Blocking somatic repeat expansion and lowering huntingtin by RNAi synergize to attenuate Huntington’s disease pathogenesis in mice

  • Jillian Belgrad1
  • Ashley Summers1
  • Christian Landles2
  • Jonathan R. Greene3
  • Samuel R. Hildebrand1
  • Emily G. Knox1
  • Ellen Sapp4
  • Nozomi Yamada1
  • Raymond C. Furgal1
  • Rachael Miller1
  • Georgina F Osborne2
  • Kathryn O. Chase1
15 more authors
  • Eric Luu1
  • Jason Freedman5
  • Brianna M. Bramato1
  • Nicholas McHugh1
  • Israel Nnah6
  • Vicky Benoit1
  • Daniel O’Reilly1
  • Paul L. Greer5
  • Gillian P. Bates2
  • Thomas Vogt7
  • Ramee Lee7
  • David Howland7
  • Marian DiFiglia4
  • Neil Aronin M. D8,1
  • Anastasia Khvorova5,1
8 affiliations
  1. 1UMass Chan Medical School, RNA Therapeutics Institute, Worcester, MA 01605, USA
  2. 2Department of Neurodegenerative Disease and Huntington’s Disease Centre, Queen Square Institute of Neurology, UCL, London WC1N 3BG, UK
  3. 3Rancho Biosciences LLC, San Diego, CA 92127, USA
  4. 4Massachusetts General Hospital, Mass General Institute for Neurodegenerative Disease (MIND), Charlestown, MA 02129, USA
  5. 5UMass Chan Medical School, Program in Molecular Medicine, Worcester, MA 01605, USA
  6. 6Charles River Laboratories Inc., 334 South Street, Shrewsbury, MA 01545, USA
  7. 7CHDI Foundation, Princeton, NJ 08540, USA
  8. 8UMass Chan Medical School, Department of Medicine, Worcester, MA 01605, USA
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Cite

BibTeXRIS
BibTeX
@article{belgrad2026blocking,
  title = {{Blocking somatic repeat expansion and lowering huntingtin by RNAi synergize to attenuate Huntington’s disease pathogenesis in mice}},
  author = {Jillian Belgrad and Ashley Summers and Christian Landles and Jonathan R. Greene and Samuel R. Hildebrand and Emily G. Knox and Ellen Sapp and Nozomi Yamada and Raymond C. Furgal and Rachael Miller and Georgina F Osborne and Kathryn O. Chase and Eric Luu and Jason Freedman and Brianna M. Bramato and Nicholas McHugh and Israel Nnah and Vicky Benoit and Daniel O’Reilly and Paul L. Greer and Gillian P. Bates and Thomas Vogt and Ramee Lee and David Howland and Marian DiFiglia and Neil Aronin M. D and Anastasia Khvorova},
  journal = {Science Translational Medicine},
  year = {2026},
  month = aug,
  doi = {10.1126/scitranslmed.aea3104},
  url = {https://doi.org/10.1101/2025.06.24.661398},
}
RIS
TY  - JOUR
TI  - Blocking somatic repeat expansion and lowering huntingtin by RNAi synergize to attenuate Huntington’s disease pathogenesis in mice
AU  - Jillian Belgrad
AU  - Ashley Summers
AU  - Christian Landles
AU  - Jonathan R. Greene
AU  - Samuel R. Hildebrand
AU  - Emily G. Knox
AU  - Ellen Sapp
AU  - Nozomi Yamada
AU  - Raymond C. Furgal
AU  - Rachael Miller
AU  - Georgina F Osborne
AU  - Kathryn O. Chase
AU  - Eric Luu
AU  - Jason Freedman
AU  - Brianna M. Bramato
AU  - Nicholas McHugh
AU  - Israel Nnah
AU  - Vicky Benoit
AU  - Daniel O’Reilly
AU  - Paul L. Greer
AU  - Gillian P. Bates
AU  - Thomas Vogt
AU  - Ramee Lee
AU  - David Howland
AU  - Marian DiFiglia
AU  - Neil Aronin M. D
AU  - Anastasia Khvorova
PY  - 2026
DA  - 2026/08/12
JO  - Science Translational Medicine
DO  - 10.1126/scitranslmed.aea3104
UR  - https://doi.org/10.1101/2025.06.24.661398
SN  - 1946-6234
SN  - 1946-6242
ER  -