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

Cheerie, David; Meserve, Margaret M.; Beijer, Danique; Kaiwar, Charu; Newton, Logan; Tavares, Ana Lisa Taylor; Verran, Aubrie Soucy; Sherrill, Emma; Leonard, Stefanie; Sanders, Stephan J.; Blake, Emily; Elkhateeb, Nour; Gandhi, Aastha; Liang, Nicole S. Y.; Morgan, Jack T.; Verwillow, Anna; Verheijen, Jan; Giles, Andrew; Williams, Sean; Chopra, Maya; Croft, Laura; Dafsari, Hormos Salimi; Davidson, Alice E.; Friedman, Jennifer; Gregor, Anne; Haque, Bushra; Lechner, Rosan; Montgomery, Kylie-Ann; Ryten, Mina; Schober, Emil; Siegel, Gabriele; Sullivan, Patricia J.; Whittle, Ella F.; Zardetto, Bianca; Yu, Timothy W.; Synofzik, Matthis; Aartsma-Rus, Annemieke; Costain, Gregory; Lauffer, Marlen C.

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

Author:

Cheerie, David; Meserve, Margaret M.; Beijer, Danique; Kaiwar, Charu; Newton, Logan; Tavares, Ana Lisa Taylor; Verran, Aubrie Soucy; Sherrill, Emma; Leonard, Stefanie; Sanders, Stephan J.; Blake, Emily; Elkhateeb, Nour; Gandhi, Aastha; Liang, Nicole S. Y.; Morgan, Jack T.; Verwillow, Anna; Verheijen, Jan; Giles, Andrew; Williams, Sean; Chopra, Maya; Croft, Laura; Dafsari, Hormos Salimi; Davidson, Alice E.; Friedman, Jennifer; Gregor, Anne; Haque, Bushra; Lechner, Rosan; Montgomery, Kylie-Ann; Ryten, Mina; Schober, Emil; Siegel, Gabriele; Sullivan, Patricia J.; Whittle, Ella F.; Zardetto, Bianca; Yu, Timothy W.; Synofzik, Matthis; Aartsma-Rus, Annemieke; Costain, Gregory; Lauffer, Marlen C.

Tübinger Autor(en):

Beijer, Danique
Synofzik, Matthis

Published in:

American Journal of Human Genetics (2025), Bd. 112, H. 5, S. 975-983

Verlagsangabe:

Cambridge : Cell Press

Language:

English

Full text:

http://dx.doi.org/10.1016/j.ajhg.2025.02.017

ISSN:

0002-9297