首页>
外国专利>
Spliceosome-mediated RNA trans-splicing, and modifications of the genetic defect of factor VIII using spliceosome-mediated RNA trans-splicing
Spliceosome-mediated RNA trans-splicing, and modifications of the genetic defect of factor VIII using spliceosome-mediated RNA trans-splicing
展开▼
机译:剪接体介导的RNA转拼,以及使用剪接体介导的RNA转拼修饰因子VIII的遗传缺陷
展开▼
页面导航
摘要
著录项
相似文献
摘要
The compositions of the invention include pre-trans-splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans-splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA). In particular, the PTMs of the present invention are genetically engineered to interact with factor VIII (FVIII) target pre-mRNA so as to result in correction of clotting FVIII genetic defects responsible for hemophilia A. The compositions of the invention further include recombinant vector 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 a FVIII target pre-mRNA under conditions in which a portion of the PTM is trans-spliced to a portion of the target pre-mRNA to form a RNA molecule wherein the genetic defect in the FVIII gene has been corrected. The methods and compositions of the present invention can be used in gene therapy for correction of FVIII disorders such as hemophilia A.
展开▼