US2014243264A1PendingUtilityA1
Methods of treatment for retinal diseases
Est. expiryJun 9, 2031(~4.9 yrs left)· nominal 20-yr term from priority
Inventors:Rong Wen
A61P 27/06A61P 25/28A61P 27/02C07K 14/475A61K 38/18
47
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Claims
Abstract
The present invention provides methods of treating a retinal disorder comprising administering an effective amount of a neurotrophic factor to a subject having the retinal disorder. The neurotrophic factors useful in the invention include mesencephalic astrocyte-derived neurotrophic factor (MANF) and conserved dopamine neurotrophic factor (CDNF). The present invention further comprises pharmaceutical compositions and kits containing MANF and CDNF.
Claims
exact text as granted — not AI-modifiedWhat is claimed is:
1 . A method of treating a retinal disorder, said method comprising administering an effective amount of MANF to a subject with said retinal disorder.
2 . The method of claim 1 , wherein said MANF comprises the amino acid sequence of SEQ ID NO: 3, or a fragment thereof.
3 . The method of claim 1 , wherein said retinal disorder is a neurodegenerative retinal disorder.
4 . The method of claim 1 , further comprising administering an effective amount of CDNF.
5 . The method of claim 4 , wherein said CDNF comprises the amino acid sequence of SEQ ID NO: 4, or a fragment thereof.
6 . The method of claim 1 , wherein MANF is in a pharmaceutically acceptable carrier.
7 . The method of claim 6 , wherein the pharmaceutically acceptable carrier is a saline solution.
8 . The method of claim 1 , wherein MANF is administered to an eye of said subject.
9 . The method of claim 8 , wherein administering occurs by injection.
10 . The method of claim 9 , wherein said injection is an intravitreal injection.
11 . The method of claim 1 , wherein MANF is administered in an area adjacent to the eye.
12 . The method of claim 1 , wherein said retinal disorder is a result of injury to a tissue or a cell of the central nervous system.
13 . The method of claim 12 , wherein said tissue or said cell of the central nervous system is a ganglion cell.
14 . The method of claim 12 , wherein said tissue or said cell of the central nervous system is a photoreceptor cell.
15 . The method of claim 1 , wherein said retinal disorder is selected from the group consisting of a genetic disorder and a sporadic disorder.
16 . A method for promoting neuroprotection in a neuronal cell, comprising contacting said neuronal cell with a neurotrophic factor selected from the group consisting of CDNF, MANF, and combinations thereof.
17 . The method of claim 16 , wherein said contacting occurs in vivo.
18 . The method of claim 16 , wherein said contacting occurs in vitro.
19 . The method of claim 16 , wherein said neuronal cell is a retinal ganglion cell.
20 . The method of claim 16 , wherein said neuronal cell is a photoreceptor cell.
21 . The method of claim 16 , wherein said neurotrophic factor is a recombinant neurotrophic factor.
22 . The method of claim 16 , wherein said neurotrophic factor is a human neurotrophic factor.
23 . The method of claim 22 , wherein said human neurotrophic factor is a recombinant human neurotrophic factor.
24 . The method of claim 16 , wherein said MANF comprises the amino acid sequence of SEQ ID NO: 3, or a fragment thereof.
25 . The method of claim 16 , wherein said CDNF comprises the amino acid sequence of SEQ ID NO: 4, or a fragment thereof.
26 . A pharmaceutical composition comprising a neurotrophic factor selected from the group consisting of MANF, CDNF and combinations thereof.
27 . The method of claim 26 , wherein said MANF comprises the amino acid sequence of SEQ ID NO: 3, or a fragment thereof.
28 . The method of claim 26 , wherein said CDNF comprises the amino acid sequence of SEQ ID NO: 4, or a fragment thereof.
29 . The pharmaceutical composition of claim 26 , further comprising an aqueous solution and one or more pharmaceutically acceptable excipients, additives, carriers or adjuvants.
30 . A method of treating a retinal disorder, said method comprising administering an effective amount of CDNF to a subject with said retinal disorder.
31 . The method of claim 30 , wherein said CDNF comprises the amino acid sequence of SEQ ID NO: 4, or a fragment thereof.
32 . The method of claim 30 , further comprising administering an effective amount of MANF.
33 . The method of claim 32 , wherein said MANF comprises the amino acid sequence of SEQ ID NO: 3, or a fragment thereof.
34 . The method of claim 30 , wherein said retinal disorder is a neurodegenerative retinal disorder.
35 . The method of claim 30 , wherein CDNF is in a pharmaceutically acceptable carrier.
36 . The method of claim 35 , wherein the pharmaceutically acceptable carrier is a saline solution.
37 . The method of claim 30 , wherein CDNF is administered to an eye of said subject.
38 . The method of claim 37 , wherein administering occurs by injection.
39 . The method of claim 38 , wherein said injection is an intravitreal injection.
40 . The method of claim 30 , wherein CDNF is administered in an area adjacent to the eye.
41 . The method of claim 30 , wherein said retinal disorder is a result of injury to a tissue or a cell of the central nervous system.
42 . The method of claim 41 , wherein said tissue or said cell of the central nervous system is a ganglion cell.
43 . The method of claim 41 , wherein said tissue or said cell of the central nervous system is a photoreceptor cell.
44 . The method of claim 30 , wherein said retinal disorder is selected from the group consisting of a genetic disorder and a sporadic disorder.
45 . A kit comprising a neurotrophic factor selected from the group consisting of MANF, CDNF and combinations thereof; one or more reagents; and instructions for use thereof.
46 . The kit of claim 45 , wherein said MANF comprises the amino acid sequence of SEQ ID NO: 3, or a fragment thereof.
47 . The kit of claim 45 , wherein said CDNF comprises the amino acid sequence of SEQ ID NO: 4, or a fragment thereof.Cited by (0)
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