Synthetic polynucleotides encoding a human FKRP protein
US11596698B2 · kind B2 · utility
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Key dates
| Filing date | Jul 6, 2018 |
| Grant date | Mar 7, 2023 |
| Priority date | — |
| Expiry date | Aug 21, 2038 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2750/14143
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention concerns synthetic polynucleotides encoding a human fukutin-related protein (FKRP) wherein the synthetic polynucleotides contain at least a mutation avoiding supplementary transcript(s) generated from frameshift start codon(s). The synthetic polynucleotides are useful, especially for treating a pathology linked to a FKRP deficiency or induced by a defect in α-dystroglycan (α-DG) glycosylation, such as LGMD2I.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.