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Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments.
Cheerie D, Meserve MM, Beijer D, Kaiwar C, Newton L, Taylor Tavares AL, Verran AS, Sherrill E, Leonard S, Sanders SJ, Blake E, Elkhateeb N, Gandhi A, Liang NSY, Morgan JT, Verwillow A, Verheijen J, Giles A, Williams S, Chopra M, Croft L, Dafsari HS, Davidson AE, Friedman J, Gregor A, Haque B, Lechner R, Montgomery KA, Ryten M, Schober E, Siegel G, Sullivan PJ, Whittle EF, Zardetto B, Yu TW, Synofzik M, Aartsma-Rus A, Costain G, Lauffer MC; N=1 Collaborative. Cheerie D, et al. Among authors: meserve mm. Am J Hum Genet. 2025 May 1;112(5):975-983. doi: 10.1016/j.ajhg.2025.02.017. Epub 2025 Mar 25. Am J Hum Genet. 2025. PMID: 40139194 Free PMC article. Review.
Screening rare genetic diagnoses for amenability to bespoke antisense oligonucleotide therapy development: A retrospective cohort study.
Cheerie D, Lauffer MC, Newton L, Amburgey K, Beijer D, Haque B, Kalish BT, Meserve MM, Oh RY, Pan AY, Reuter MS, Szego MJ, Szuto A; N=1 Collaborative; Aartsma-Rus A, Axford MM, Deshwar AR, Dowling JJ, Marshall CR, Ivakine Z, Synofzik M, Yu TW, Costain G. Cheerie D, et al. Among authors: meserve mm. Genet Med. 2025 Oct 11;28(1):101597. doi: 10.1016/j.gim.2025.101597. Online ahead of print. Genet Med. 2025. PMID: 41090344