Compositions and methods for treatment of mitochondrial diseases
View Patent ↗Methods and compositions are provided for the treatment of a mitochondrial disease in an individual with the mitochondrial disease. Aspects of the methods include administering an inhibitor of a mitochondrial transport protein to a subject having a mitochondrial disease. Also provided are reagents and kits for practicing the subject methods.
1. A method of treating mitochondrial disease in a subject having a mitochondrial disease, comprising
administering a therapeutically effective amount of an inhibitor of a mitochondrial transport protein to the subject, wherein the administering stabilizes or reduces an undesirable clinical symptom in the subject.
2. The method according to claim 1 , wherein the mitochondrial transport protein is a Miro protein.
3. The method according to claim 1 , wherein the mitochondrial transport protein is a trafficking kinesin (TRAK) protein.
4. The method according to claim 1 , wherein the mitochondrial transport protein is kinesin heavy chain (Khc).
5. The method according to claim 1 , wherein the inhibitor is a nucleic acid.
6. The method according to claim 1 , wherein the mitochondrial disease is Parkinson's disease.
7. The method according to claim 6 , wherein the Parkinson's disease is a PTEN-induced putative kinase 1 (PINK-1)-associated form of PD.
8. The method according to claim 6 , wherein the inhibitor is administered to the midbrain and/or putamen of the subject.
9. The method according to claim 6 , further comprising the step of administering to the individual a therapeutically effective amount of one or more agents selected from the group consisting of levodopa, a dopamine agonist, a MAO-B inhibitor, amantadine, or an anticholinergic prior to, concurrently with, or after administering the inhibitor of a mitochondrial transport protein.
10. A method of treating Parkinson's disease in a subject having Parkinson's disease, comprising:
administering a therapeutically effective amount of an inhibitor of a Miro protein to the subject having Parkinson's disease (PD), wherein the administering stabilizes or reduces an undesirable clinical symptom in the subject,
wherein the Parkinson's disease is a PTEN-induced putative kinase 1 (PINK-1)-associated form of PD.
11. The method according to claim 10 , wherein the inhibitor is a nucleic acid.
12. The method according to claim 10 , wherein the inhibitor is administered to the midbrain and/or putamen of the subject.