US Patent:
20230022489, Jan 26, 2023
Inventors:
Athma A. PAI - Cambridge MA, US
Jonathan K. WATTS - Worcester MA, US
Kaitlyn VALLA - Worcester MA, US
Eraj Shafiq KHOKHAR - Worcester MA, US
Zachary KARTJE - Worcester MA, US
International Classification:
C12N 15/113
C12Q 1/6834
A61P 43/00
Abstract:
The present disclosure provides methods of identifying non-productive splice sites in target RNA transcripts and antisense oligonucleotides that increase the expression of said target RNA transcripts. In an embodiment, the target RNA transcript comprises ADAR, ARSA, ATPIA2, CACNAIA, DNMI, EIF2BI, EIF2B2, EIF2B5, IDUA, MFSD8, NF2, NPC1L PEXI, PRICKLE2, PRRT2, RAM, SETD5, SHANKS, SLC6A1, STXBPI, STX1B, and TCF4.