Pre-MRNA trans-splicing molecule (RTM) molecules and their uses
US8735366B2 · kind B2 · utility
Inventors
Key dates
| Filing date | Jul 30, 2009 |
| Grant date | May 27, 2014 |
| Priority date | — |
| Expiry date | Oct 12, 2030 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2840/445
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
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.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.