Consensus guidelines for eligibility assessment of pathogenic variants to antisense oligonucleotide treatments

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Authors: David Cheerie, Margaret 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 Sullivan, Bianca Zardetto, Timothy Yu, Matthis Synofzik, Annemieke Aartsma-Rus, Gregory Costain, Marlen C. Lauffer

Journal publisher: Cold Spring Harbor Laboratory

Published year: 2025

DOI identifier: 10.1101/2024.09.27.24314122

ISSN: 1537-6605