Polynucleotides encoding engineered meganucleases having specificity for recognition sequences in the dystrophin gene
US12006522B2 · kind B2 · utility
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Key dates
| Filing date | Jul 27, 2023 |
| Grant date | Jun 11, 2024 |
| Priority date | — |
| Expiry date | Jul 27, 2043 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12Y301/25
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present disclosure encompasses engineered meganucleases that bind and cleave recognition sequences within a dystrophin gene. The present disclosure also encompasses methods of using such engineered meganucleases to make genetically modified cells. Further, the disclosure encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or polynucleotides encoding engineered meganucleases of the disclosure, and the use of such compositions for the modification of a dystrophin gene in a subject, or for treatment of Duchenne Muscular Dystrophy.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.