Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments

David Cheerie(SickKids Foundation), Marlen C. Lauffer(Leiden University Medical Center), Ana Lisa Taylor Tavares(University of Leeds), Anne Gregor(University of Bern), Ella F. Whittle(St George's, University of London), Logan Newton(University of Toronto), Nicole Si Yan Liang(University of Toronto), Matthis Synofzik(German Center for Neurodegenerative Diseases), Annemieke Aartsma‐Rus(Leiden University Medical Center), Maya Chopra(Boston Children's Hospital), Hormos Salimi Dafsari(University of Cologne), Andrew C. Giles(University of British Columbia), Jan Verheijen(ARUP Laboratories (United States)), Nour Elkhateeb(Cambridge University Hospitals NHS Foundation Trust), Kylie Montgomery(New England Biolabs (United States)), J. Morgan(Leiden University Medical Center), Alice E. Davidson(Moorfields Eye Hospital NHS Foundation Trust), Stephan Sanders(University of California, San Francisco), Gregory Costain(University of Toronto), Gabriele Siegel(University of Zurich), Patricia J. Sullivan(Cancer Institute of New South Wales), Timothy W. Yu(Boston Children's Hospital), Danique Beijer(German Center for Neurodegenerative Diseases), Laura V. Croft(Queensland University of Technology), Aubrie Soucy(Institut thématique Génétique, génomique et bioinformatique), Jennifer Friedman(Rady Children's Hospital-San Diego), Emily J. Blake(Massachusetts Institute of Technology), Bianca Zardetto(Leiden University Medical Center), Bushra Haque(SickKids Foundation), Emma Sherrill(Boston Children's Hospital), Anna Verwillow(Massachusetts General Hospital), Margaret Meserve(Boston Children's Hospital), Emil Schober(Max Planck Institute for Biology of Ageing), Stefanie Leonard(Somerville Hospital), Aastha Gandhi(University of Toronto), Mina Ryten(National Hospital for Neurology and Neurosurgery), Rosan Lechner(Erasmus MC), Sean Williams(University of Toronto), Charu Kaiwar(Mayo Clinic)
The American Journal of Human Genetics
March 26, 2025
Cited by 22


Related Papers