Consensus guidelines for eligibility assessment of pathogenic variants to antisense oligonucleotide treatments
David Cheerie, Margaret M. Meserve, Danique Beijer, Charu Kaiwar, Logan Newton, Ana Lisa Taylor Tavares, Aubrie Soucy, Emma Sherrill, Stefanie Leonard, Stephan Sanders, Emily J. Blake, Nour M. Elkhateeb, Aastha Gandhi, Nicole Si Yan Liang, J. Morgan, , Marlen C. Lauffer
The paper presents consensus guidelines for assessing pathogenic variants' eligibility for antisense oligonucleotide treatments, including classification criteria and considerations for different therapeutic approaches.
The American Journal of Human Genetics · Mar 26, 2025Peer-reviewedGenetic & Genomic Medicine