Patent ID: 9303078
Filing Date: 2016-04-05
CPC Classification: A61K,C07K,C12N

Claim Text:
1. A nucleic acid molecule comprising: a) two target binding domains AS and AS′ that target the binding of the nucleic acid molecule to a target pre-mRNA, wherein said two target binding domains AS and AS′ are located respectively at the 5′-end and at the 3′-end of the nucleic acid molecule, b) a 3′ splice region comprising a branch point, a polypyrimidine tract and a 3′ splice acceptor site, c) a 5′ splice region comprising a 5′ splice donor site, d) a spacer sequence that separates said 3′ splice region from said 5′-end target binding domain AS, e) a spacer sequence that separates said 5′ splice region from said 3′-end target binding domain AS′, said spacer comprising a downstream intronic splice enhancer (DISE) and f) a nucleotide sequence to be trans-spliced to said target pre-mRNA, wherein said nucleotide sequence encodes at least a part of a polypeptide, and is located between said 3′ splice region and said 5′ splice region of said nucleic acid.