Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations
US8802437B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Sep 24, 2010 |
| Grant date | Aug 12, 2014 |
| Priority date | — |
| Expiry date | Sep 24, 2030 |
Classification
- Technology area (CPC A)Human Necessities
- CPC primaryA61K38/00
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention relates to a method to treat a genetic disease in an individual caused by at least one frame shift or at least one non sense mutation in the human dystrophin gene comprising at least the step of bringing into contact at least one meganuclease enzyme, which recognizes and cuts a target site in the human dystrophin gene, with the genome of said individual under conditions wherein said at least one meganuclease recognizes and cleaves its target site in the human dystrophin gene. Said method applies also to a set of meganuclease enzymes, which each recognizes and cuts a different target site. The present invention also relates to a kit comprising, at least one meganuclease enzyme as defined above and medicament comprising said meganuclease.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.