Abstract
Described herein are antisense oligonucleotides (ASOs), vectors, and related compositions and methods for increasing endogenous expression of SHANK3 protein and uses thereof for conditions associated with SHANK3 haploinsufficiency such as Phelan-McDermid syndrome.
Full Text
What is claimed is:
Described herein are antisense oligonucleotides (ASOs), vectors, and related compositions and methods for increasing endogenous expression of SHANK3 protein and uses thereof for conditions associated with SHANK3 haploinsufficiency such as Phelan-McDermid syndrome.
Timeline
Filed
04/01/2026Published
07/30/2026Granted
Not AvailableIPC Codes(3)
C12N 15/113:Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 31/7088:Compounds having three or more nucleosides or nucleotides
A61P 25/28:for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia