Specific targeting of RNA expanded repeat sequences
US9933419B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Feb 8, 2017 |
| Grant date | Apr 3, 2018 |
| Priority date | — |
| Expiry date | Feb 8, 2037 |
Classification
- Technology area (CPC G)Physics
- CPC primaryG01N2500/10
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention provides small molecule compounds that can form covalent adducts with specific sequences of RNA, such as the hairpin loop r(CUG)exp sequence which is a cause of myotonic dystrophy type 1 (DM1), or the r(CGG)exp sequence which is a cause of fragile X-associated tremor/ataxia syndrome (FXTAS); to methods of making the small molecule compounds; and to methods of using the small molecular compounds in the treatment of DM1 or of FXTAS in patients afflicted therewith. The invention further provides a method for identifying an RNA target of a small molecule drug in vivo, using a small molecule drug conjugated to an RNA-reactive crosslinker group and a reporter group, contacting a cell or nucleic acid extract with the small molecule drug conjugate, then separating RNA targets crosslinked to the small molecule drug conjugate by interaction of the affinity group with a complementary affinity group.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.