Abstract
Disclosed herein are systems for modifying nucleic acid splicing of a target RNA. The system may be useful in a method such as a method for treating a genetic disorder such as epilepsy. The system may include an expression system, or a modified small nuclear RNA (snRNA) that includes an exonic splicing silencer (ESS) sequence, and a target nucleic acid sequence that targets the target RNA. Described herein are recombinant regulatory elements for expression systems. Also described are nucleic acid expression systems with recombinant regulatory elements.
Full Text
What is claimed is:
Disclosed herein are systems for modifying nucleic acid splicing of a target RNA. The system may be useful in a method such as a method for treating a genetic disorder such as epilepsy. The system may include an expression system, or a modified small nuclear RNA (snRNA) that includes an exonic splicing silencer (ESS) sequence, and a target nucleic acid sequence that targets the target RNA. Described herein are recombinant regulatory elements for expression systems. Also described are nucleic acid expression systems with recombinant regulatory elements.
Timeline
Filed
04/01/2026Published
07/23/2026Granted
Not AvailableIPC Codes(3)
C12N 15/85:for animal cells
C12N 15/113:Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12N 15/67:General methods for enhancing the expression