diranersen (BIIB080) - Biogen
Alternative splicing in Alzheimer's disease: event-level evidence, risk-locus biology, and RNA-based therapeutic perspectives. (PubMed, Gene) - Sep 16, 2026 - "ApoER2 exon 19 correction provides AD-specific preclinical evidence for splice-event modulation, whereas BIIB080/MAPTRx illustrates the feasibility of CNS antisense oligonucleotide delivery and target-engagement monitoring in patients...Overall, AS should not be viewed as a single pathogenic mechanism in AD. Selected splice events may improve AD risk-locus interpretation, molecular stratification, biomarker development, and the design of RNA-based therapeutic strategies." 
Journal • Review • Alzheimer's Disease • CNS Disorders • APOE • CD33 • MAPT • TARDBP
http://www.ncbi.nlm.nih.gov/pubmed/42744164
 
Daoyuan Yue; Jinglin Li; Wenge Xie; Xiong Wang; Zhigang Xiong
 
Sep 16, 2026
 
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