Diagnosis and treatment of friedreich's ataxia
US9476043B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Apr 4, 2012 |
| Grant date | Oct 25, 2016 |
| Priority date | — |
| Expiry date | May 27, 2032 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12Q2600/156
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention is directed to oligonucleotides based on peptide nucleic acid oligonucleotide or an equivalent oligonucleotide analogue, such as morpholino or a locked nucleic acid sequences and the use of such oligonucleotides for the dissociation of higher order structures, including triplex-helix DNA structures, in repeated sequences of DNA in Friedreich's ataxia. The dissociation of such structures may be used in the diagnosis and/or treatment of Friedreich's ataxia. Consequently, the present invention is also directed to a method for diagnosing Friedreich's ataxia and the use of peptide nucleic acid oligonucleotide or an equivalent oligonucleotide analogue, such as morpholino or a locked nucleic acid sequences in the treatment of Friedreich's ataxia. Preferably, the oligonucleotides comprise a sequence selected from the group consisting of (GAA)n, (CTT)n, (JTT)n or a mixed (JTT/CTT)n sequence.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.