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

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Date: May 15, 2025
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
Journal: American Journal of Human Genetics