Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments
Authors: David Cheerie; Margaret M Meserve; Danique Beijer; Charu Kaiwar; Logan Newton; Ana Lisa; Taylor Tavares; Aubrie Soucy Verran; Emma Sherrill; Stefanie Leonard; Stephan J Sanders; Emily Blake; Nour Elkhateeb; Aastha Gandhi; Nicole S Y Liang; Jack T Morgan; Anna Verwillow; Jan Verheijen; Andrew Giles; Sean Williams; Maya Chopra; Laura Croft; Hormos Salimi Dafsari; Alice E Davidson; Jennifer Friedman; Anne Gregor; Bushra Haque; Rosan Lechner; Kylie-Ann Montgomery; Mina Ryten; Emil Schober; Gabriele Siegel; Patricia J Sullivan; Ella F Whittle; Bianca Zardetto; Timothy W Yu; Matthis Synofzik; Annemieke Aartsma-Rus; Gregory Costain; Marlen C Lauffer
Collaborators: Boston Children's Hospital; Cambridge University Hospitals; Erasmus Medical Centre; Harvard Medical School; Inselspital University Hospital Bern; King's College London; Leiden University Medical Center; Massachusetts General Hospital; Mayo Clinic; Queensland Health; The Hospital for Sick Children, The University of Toronto; University of California, San Diego; University of Cologne; University of Colorado School of Medicine; University of Oxford; University of Tübingen; University of Utah; University of Zurich; UNSW Sydney