Targeted pre-mRNA/mRNA modification and gene regulation
US9238813B2 · kind B2 · utility
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19Claims
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Key dates
| Filing date | Oct 17, 2013 |
| Grant date | Jan 19, 2016 |
| Priority date | — |
| Expiry date | Oct 17, 2033 |
Classification
- Technology area (CPC A)Human Necessities
- CPC primaryA61K48/00
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Methods for affecting mRNA expression or translation through the modification of pre-mRNA or mRNA transcripts are described. In one embodiment of the methods of the present invention, the branch point adenosine of a pre-mRNA transcript is 2′-0-methylated to block splicing and subsequent expression of the protein encoded by the transcript. In another embodiment, a uridine residue in a nonsense stop codon may be modified to pseudouridine, causing the translation machinery to read through the nonsense stop codon and translate a full length protein.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.