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.

dc.contributor.author	Beijer, Danique
dc.contributor.author	Synofzik, Matthis
dc.date.accessioned	2025-09-19T14:48:40Z
dc.date.available	2025-09-19T14:48:40Z
dc.date.issued	2025
dc.identifier.issn	0002-9297
dc.identifier.uri	http://hdl.handle.net/10900/170345
dc.language.iso	en	de_DE
dc.publisher	Cambridge : Cell Press	de_DE
dc.relation.uri	http://dx.doi.org/10.1016/j.ajhg.2025.02.017	de_DE
dc.subject.ddc	570	de_DE
dc.subject.ddc	610	de_DE
dc.title	Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments	de_DE
dc.type	Article	de_DE
utue.quellen.id	20250630000000_00609
utue.publikation.seiten	975-983	de_DE
utue.personen.roh	Cheerie, David
utue.personen.roh	Meserve, Margaret M.
utue.personen.roh	Beijer, Danique
utue.personen.roh	Kaiwar, Charu
utue.personen.roh	Newton, Logan
utue.personen.roh	Tavares, Ana Lisa Taylor
utue.personen.roh	Verran, Aubrie Soucy
utue.personen.roh	Sherrill, Emma
utue.personen.roh	Leonard, Stefanie
utue.personen.roh	Sanders, Stephan J.
utue.personen.roh	Blake, Emily
utue.personen.roh	Elkhateeb, Nour
utue.personen.roh	Gandhi, Aastha
utue.personen.roh	Liang, Nicole S. Y.
utue.personen.roh	Morgan, Jack T.
utue.personen.roh	Verwillow, Anna
utue.personen.roh	Verheijen, Jan
utue.personen.roh	Giles, Andrew
utue.personen.roh	Williams, Sean
utue.personen.roh	Chopra, Maya
utue.personen.roh	Croft, Laura
utue.personen.roh	Dafsari, Hormos Salimi
utue.personen.roh	Davidson, Alice E.
utue.personen.roh	Friedman, Jennifer
utue.personen.roh	Gregor, Anne
utue.personen.roh	Haque, Bushra
utue.personen.roh	Lechner, Rosan
utue.personen.roh	Montgomery, Kylie-Ann
utue.personen.roh	Ryten, Mina
utue.personen.roh	Schober, Emil
utue.personen.roh	Siegel, Gabriele
utue.personen.roh	Sullivan, Patricia J.
utue.personen.roh	Whittle, Ella F.
utue.personen.roh	Zardetto, Bianca
utue.personen.roh	Yu, Timothy W.
utue.personen.roh	Synofzik, Matthis
utue.personen.roh	Aartsma-Rus, Annemieke
utue.personen.roh	Costain, Gregory
utue.personen.roh	Lauffer, Marlen C.
dcterms.isPartOf.ZSTitelID	American Journal of Human Genetics	de_DE
dcterms.isPartOf.ZS-Issue	5	de_DE
dcterms.isPartOf.ZS-Volume	112	de_DE
utue.fakultaet	04 Medizinische Fakultät	de_DE