Compositions and methods for treating leber's hereditary optic neuropathy
AssigneeWuhan Neurophth Biotechnology 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.
Claims47
A method of treating an eye disorder, comprising administering to a patient in need thereof
- a) a first pharmaceutical composition comprising an adeno-associated virus (AAV) comprising a recombinant nucleic acid comprising:
- i) a nucleic acid sequence encoding a mitochondrial targeting peptide;
- ii) a nucleic acid sequence encoding a mitochondrial protein comprising a nucleic acid sequence that is at least 90%, at least 95%, at least 97%, at least 99%, or 100% identical to a sequence selected from the group consisting of SEQ ID NO:
- 6-7 and 9-12;
- and
- iii) a 3′UTR nucleic acid sequence;
- and
- b) a second pharmaceutical composition comprising a steroid.
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A method of screening patients for treatment of an eye disorder, the method comprising:
- (a) obtaining a serum sample from a patient;
- (b) culturing a population of target cells with a composition comprising an adeno-associated virus (AAV) comprising a recombinant nucleic acid encoding a detectable label in the presence of the serum sample;
- and
- (c) detecting the expression level of the detectable label in the target cell population after the culturing, wherein the patient is selected for the treatment if the expression level of the detectable label in the target cell population is higher than a pre-determined threshold.
A method of treating an eye disorder for a patient in need thereof, comprising:
- (a) obtaining a serum sample from a patient;
- (b) culturing a population of target cells with a composition comprising a first adeno-associated virus (AAV) comprising a first recombinant nucleic acid encoding a detectable label in the presence of the serum sample;
- (c) detecting the expression level of the detectable label in the target cell population;
- and
- (d) administering to the patient a pharmaceutical composition comprising a second AAV comprising a second recombinant nucleic acid, wherein the expression level of the detectable label in the target cell population is higher than a pre-determined threshold.
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A method of treating Leber's hereditary optic neuropathy (LHON), comprising administering to a patient in need thereof:
- (a) a first pharmaceutical composition comprising an adeno-associated virus (AAV) comprising a recombinant nucleic acid comprising:
- i) a nucleic acid sequence encoding a mitochondrial targeting peptide comprising an amino sequence that is at least 90%, at least 95%, at least 97%, at least 99%, or 100% identical to a sequence selected from the group consisting of SEQ ID NO:
- 126-159;
- ii) a nucleic acid sequence encoding a mitochondrial protein comprising a nucleic acid sequence that is at least 90%, at least 95%, at least 97%, at least 99%, or 100% identical to a sequence selected from the group consisting of SEQ ID NO:
- 6-7 and 9-12;
- and
- iii) a 3′UTR nucleic acid sequence that is at least 90%, at least 95%, at least 97%, at least 99%, or 100% identical to a sequence selected from the group consisting of SEQ ID NO:
- 13, 14, and 111-125;
- and
- (b) a second pharmaceutical composition comprising methylprednisolone or prednisone.
Dependencies are inferred from the claim text.