Patent ID: 8735366
Filing Date: 2014-05-27
Classification: A61K,A61P,C07K,C12N

Abstract:
1. A pre-mRNA trans-splicing molecule (RTM), comprising a) at least one binding domain that targets binding of the molecule to a pre-mRNA expressed within a cell, wherein said binding domain consists of a sequence complementary to an exonic sequence of a mammalian gene; b) at least one splicing domain containing motifs necessary for a trans-splicing reaction to occur, and c) at least one coding domain, wherein said coding domain encodes at least one exon of a mammalian gene selected from the group consisting of cystic fibrosis transmembrane conductance regulator (CFTR), integrins, TNF-alpha, interleukins, the immunoglobulin superfamily, kallikreins, matrix metalloproteinases, keratins, collagens, and laminins.