Methods and compositions for modification of splicing of pre-mRNA,申请号US20030672501-传众专利搜索

Methods and compositions for modification of splicing of pre-mRNA

申请公布号：US2004137472(A1)

申请号：US20030672501

申请日期：2003.09.26

申请公布日期：2004.07.15

申请人：

KOLE RYSZARD

发明人：KOLE RYSZARD

分类号：A61K;C12N15/63;C12P19/34;C12Q1/68;G01N33/68;(IPC1-7):C12Q1/68

主分类号：A61K

摘要：The present invention provides a method of preventing a splicing event in a pre-mRNA molecule, comprising contacting the pre-mRNA and/or elements of the splicing machinery with a small molecule compound identified according to the methods described herein to prevent the splicing event in the pre-mRNA molecule. Further provided is a method of inducing a splicing event in a pre-mRNA molecule, comprising contacting the pre-mRNA and/or elements of the splicing machinery with a small molecule compound identified according to the methods described herein to induce the splicing event in the pre-mRNA molecule. Furthermore, a method is provided herein of treating a patient having a disorder associated with an alternative or aberrant splicing event in a pre-mRNA molecule, comprising administering to the patient a therapeutically effective amount of a compound identified according to the methods described herein to prevent an alternative or aberrant splicing event in a pre-mRNA molecule, thereby treating the patient.