Document is current
Any future updates will be listed below
-
Authors
Amado, Defne A. https://orcid.org/0000-0001-6202-3900
Robbins, Ashley B.
Whiteman, Katherine R.
Smith, Alicia R.
Chillon, Guillem https://orcid.org/0000-0003-2750-9037
Chen, Yonghong
Fuller, Joshua A. https://orcid.org/0009-0004-0618-2113
Patty, Nicholas A. https://orcid.org/0009-0000-1240-1002
Izda, Aleksandar
Cheng, Congsheng
Nelson, Shareen
Dichter, Abigail I. https://orcid.org/0000-0003-3797-4987
Mazzoni, Esteban O.
Monteys, Alex Mas
Davidson, Beverly L. https://orcid.org/0000-0002-3715-6290
-
Funding
Funding for this research was provided by:
U.S. Department of Health & Human Services | NIH | National Institute of Neurological Disorders and Stroke (K08 NS114106, UH3 NS094355)
Children's Hospital of Philadelphia Research Institute
- License Information
-
More Information
Article History
Received: 12 November 2024
Accepted: 23 May 2025
First Online: 25 June 2025
Competing interests
: Y.H.C. and B.L.D. are founders of Latus Bio and have stock options. B.L.D. has sponsored research or serves an advisory role for Carbon Bio, Resilience, Roche, and Latus Bio. The other authors declare they have no competing interests. Two patents have been submitted by the Children’s Hospital of Philadelphia: PCT/US2023/071845, ‘MIRNAS Targeting ATXN2 for the Treatment of ALS and SCA2,’ D.A.A., A.M.M., and B.L.D., covering the RNAi sequences used in this manuscript; and PCT/US2020/061464, ‘Adeno-Associated Viral Vector Variants,’ Y.C. and B.L.D., covering the AAV capsid used in this manuscript.
