专利快速检索-快速检索全球专利，免费商用专利数据库-IPRDB

1. 发明申请

WO2004006678A1 SPLICEOSOME MEDIATED RNA TRANS-SPLICING FOR CORRECTION OF SKIN DISORDERS 审中-公开
标题翻译： SPLICEOSOME MEDIATED RNA TRANS-SPLICING FOR Corrients of Skine Disorders
公开(公告)号：WO2004006678A1
公开(公告)日：2004-01-22
申请号：PCT/US2003/022469
申请日：2003-07-17
申请人： INTRONN, INC. , MITCHELL, Lloyd, G. , PUTTARAJU, Madaiah , DALLINGER, Guenter , KLAUSEGGER, Alfred , BAUER, Johann
发明人： MITCHELL, Lloyd, G. , PUTTARAJU, Madaiah , DALLINGER, Guenter , KLAUSEGGER, Alfred , BAUER, Johann
IPC分类号： A01N47/40
CPC分类号： C12N15/113 , A61K48/00 , C07H21/02 , C07H21/04 , C12N2510/00
摘要： The present invention provides methods and compositions for generating novel nucleic acid molecules through targeted spliceosomal mediated RNA traps-splicing. The compositions of the invention include pre-traps-splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and mediate a traps-splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA). In particular, the PTMs of the present invention can be genetically engineered to interact with a specific target premRNA expressed in cells of the skin so as to result in correction of genetic defects responsible for a variety of different skin disorders to encode a reporter molecule or protein that may have therapeutic value. The compositions of the invention further include recombinant vectors systems capable of expressing the PTMs of the invention and cells expressing said PTMs. The methods of the invention encompass contacting the PTMs of the invention with specific target pre-Mrna expressed within cells of the skin under conditions in which a portion of the PTM is traps-spliced to a portion of the target pre-mRNA to form a chimeric RNA molecule wherein the genetic defect in the specific gene has been corrected. The present invention is based on the successful trans -splicing of the collagen XVII pre-mRNA thereby establishing the usefulness of trans -splicing for correction of skin specific genetic defects. The methods and compositions of the present invention can be used in gene therapy for treatment of specific disorders of the skin, i.e ., genodermatoses, such as epidermal fragility disorders, keratinization disorders, hair disorders and pigmentation disorders as well as cancers of the skin.
摘要翻译：本发明提供了通过靶向剪接体介导的RNA捕获 - 剪接产生新的核酸分子的方法和组合物。本发明的组合物包括设计成与靶前体信使RNA分子（靶前体mRNA）相互作用的诱捕前剪接分子（PTM），并介导产生新型嵌合RNA分子的陷阱剪接反应（嵌合 RNA）。特别地，本发明的PTM可以进行遗传工程改造以与皮肤细胞中表达的特异性靶基因前体RNA相互作用，从而导致对多种不同皮肤病症负责编码报告分子或蛋白质的遗传缺陷的校正这可能有治疗价值。本发明的组合物还包括能够表达本发明的PTM和表达所述PTM的细胞的重组载体系统。本发明的方法包括使本发明的PTM与在皮肤细胞内表达的特异性靶前Mrna接触，其中一部分PTM被剪接到目标前mRNA的一部分以形成嵌合体其中特异性基因的遗传缺陷已被校正的RNA分子。本发明基于胶原XVII前体mRNA的成功的转拼，从而确定了用于修复皮肤特异性遗传缺陷的转拼的有用性。本发明的方法和组合物可用于治疗皮肤特异性病症的基因疗法，即皮肤病，如表皮脆性障碍，角质化病症，毛发病症和色素沉着病以及皮肤癌。

IPRDB

热门服务

关于我们

友情链接

联系方式