Document is current
Any future updates will be listed below
-
Authors
Nakayama, Tojo
El Achkar, Christelle M.
Burbano, Lisseth E.
Quraishi, Imran H. https://orcid.org/0000-0003-2859-7446
Wu, Jing https://orcid.org/0000-0003-3479-5561
Li, Melody
Asami, Yutaro https://orcid.org/0009-0007-5237-5620
Golinski, Sean R.
Sherrill, Emma
Goodlett, Benjamin D.
de Gusmao, Claudio M.
Friedman, Danielle A.
Lentucci, Claudia
Suslovitch, Victoria
Riccardi, Olivia
Faour, Kamli N. W.
Kuniholm, Ashley
Soucy Verran, Aubrie https://orcid.org/0000-0001-8633-0032
Coffman, Siobhan
Ahtam, Banu
Zhao, Boxun https://orcid.org/0000-0003-2337-5756
Chin, Diana H. https://orcid.org/0000-0002-1645-3552
DiDonato, Renata L.
Hu, Chunguang A.
Lopez, Edith https://orcid.org/0009-0007-5316-8869
Hills, Sonia
Maljevic, Snezana https://orcid.org/0000-0003-1876-5872
Tran, Helene
Bush, Lynn W. https://orcid.org/0000-0001-6668-188X
Grant, Patricia Ellen
Madsen, Joseph R. https://orcid.org/0009-0001-5377-8997
Smith, Richard S. https://orcid.org/0000-0003-1937-6529
Kaczmarek, Leonard K. https://orcid.org/0000-0001-5128-6326
Berde, Charles B.
Yu, Timothy W. https://orcid.org/0000-0003-2988-7701
- License Information
-
More Information
Article History
Received: 30 August 2024
Accepted: 26 February 2026
First Online: 14 April 2026
Competing interests
: T.W.Y. has served as an ad hoc scientific consultant to and/or has received speaker honoraria from Biomarin, GeneTx, Alnylam, SynaptixBio, Third Rock Ventures, Servier Pharmaceuticals and Lilly Pharmaceuticals. He is named as an inventor on three patents pertaining to ASO treatments: for transposon-associated diseases (WO2019055460A1), ataxia telangiectasia (US20230174979A1) and progranulin (US11359199B2). He also serves as a board member of the N=1 Collaborative, the Oligonucleotide Therapeutics Society and the Society for RNA Therapeutics. He is a member of the N=1 Task Force for IRDiRC and also serves as a volunteer advisor to several nonprofit rare disease foundations. H.T. is an employee of R&D Servier Institute Paris-Saclay. The other authors declare no competing interests.
