NOTCH3 antisense oligonucleotide

NOTCH3 antisense oligonucleotide is an investigational antisense oligonucleotide (ASO) designed to target and downregulate the expression of wild-type and/or mutant NOTCH3 transcripts. Developed by UConn Health in collaboration with Ionis Pharmaceuticals, this therapeutic candidate is being evaluated for the treatment of Lateral Meningocele Syndrome (LMS), also known as Lehman Syndrome. LMS is a rare monogenic disorder caused by gain-of-function mutations in the NOTCH3 gene that lead to protein stabilization and subsequent osteopenia, neurological, and craniofacial abnormalities. By binding to NOTCH3 pre-mRNA or mRNA, the ASO induces RNase H-mediated degradation of the target transcript, thereby reducing NOTCH3 protein levels and ameliorating the gain-of-function phenotype, particularly bone loss and osteopenia.

Other names

NOTCH3 mutant ASONOTCH-3 mutant ASONOTCH 3 mutant ASONOTCH36692-93insC mutant ASONOTCH-36692-93insC mutant ASONOTCH 36692-93insC mutant ASONotch3 ASONotch-3 ASONotch 3 ASONotch36691-TAATGA mutant ASONotch-36691-TAATGA mutant ASONotch 36691-TAATGA mutant ASO