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US 2020/0263172 A1Published application
Compositions and methods for treating leber?s hereditary optic neuropathy
AssigneeWuhan Neurophth Biological Technology Limited Company
InventorBin LI
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Abstract
Disclosed herein is a recombinant nucleic acid, comprising: a mitochondrial targeting sequence; a mitochondrial protein coding sequence, wherein said mitochondrial protein coding sequence encodes a polypeptide comprising a mitochondrial protein; and a 3′UTR nucleic acid sequence. Also disclosed is a pharmaceutical composition comprising the recombinant nucleic acid and a method of treating Leber's hereditary optic neuropathy (LHON) using the pharmaceutical composition.
Claims31
1-139. (canceled)
A recombinant nucleic acid, comprising:
- a mitochondrial targeting sequence;
- a mitochondrial protein coding sequence comprising a sequence that is at least 90% identical to a sequence selected from the group consisting of SEQ ID NO:
- 7, 8, 10, and 12;
- and
- a 3′UTR nucleic acid sequence.
2 more elements
Dependencies are inferred from the claim text.