Compositions useful in treatment of ornithine transcarbamylase (OTC) deficiency
US10167454B2 · kind B2 · utility
9Cited by
53References
27Claims
0Family size
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Inventors
Key dates
| Filing date | Mar 9, 2015 |
| Grant date | Jan 1, 2019 |
| Priority date | — |
| Expiry date | Mar 9, 2035 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2800/22
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Viral vectors comprising engineered hOTC DNA and RNA sequences are provided which when delivered to a subject in need thereof are useful for treating hyperammonemia, ornithine transcarbamylase deficiency and symptoms associated therewith. Also provided are methods of using hOTC for treatment of liver fibrosis cirrhosis in OTCD patients by administering hOTC.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.