Longitudinal deep multi-omics profiling in a CLN3 Δex7/8 minipig model identifies biomarker signatures of disease

Abstract Background Development of therapies for CLN3 disease, a rare pediatric lysosomal storage disorder, has been hindered by the lack of etiological insights and translatable biomarkers to clinics. Methods We used a deep multi-omics approach to discover blood-based biomarkers using longitudinal...

Full beskrivning

Bibliografiska uppgifter

Huvudupphov:	Mitchell J. Rechtzigel, Brittany Lee, Christine Neville, Ting Huang, Ramón Díaz, Alex Rosa Campos, Khatereh Motamedchaboki, Daniel Hornburg, Tyler B. Johnson, Vicki J. Swier, Jill M. Weimer, Jon J. Brudvig
Materialtyp:	Artigo
Språk:	Inglês
Utgiven:	Nature Portfolio 2026-03-01
Serie:	Communications Medicine
Länkar:	https://doi.org/10.1038/s43856-025-01227-5
Taggar:	Lägg till en tagg Inga taggar, Lägg till första taggen!