Compositions useful in treatment of ornithine transcarbamylase (OTC) deficiency
US9890365B2 · kind B2 · utility
12Cited by
53References
15Claims
0Family size
Assignee
Inventors
Key dates
| Filing date | Mar 9, 2015 |
| Grant date | Feb 13, 2018 |
| Priority date | — |
| Expiry date | Mar 9, 2035 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2800/22
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Non-viral delivery systems 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 and/or cirrhosis in OTCD patients by administering hOTC.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.