Invention Grant
US08735366B2 Pre-MRNA trans-splicing molecule (RTM) molecules and their uses 有权
前MRNA转录分子(RTM)分子及其用途

Pre-MRNA trans-splicing molecule (RTM) molecules and their uses
Abstract:
The present invention relates to specific and markedly improved pre-mRNA trans-splicing molecule (RTM) molecules which are designed to correct specific genes expressed within cells to be targeted, and which are associated with epidermolysis bullosa, cystic fibrosis, pachyonychia congenital, and psoriasis or neurodermitis, as well as cancers of the skin. In particular, the RTMs of the present invention are genetically engineered to interact with a specific target pre-mRNA expressed in cells to be targeted so as to result in correction of genetic defects or reprogramming of gene expression responsible for a variety of different skin disorders.
Public/Granted literature
Information query
Patent Agency Ranking
0/0