Expression vector for cholesterol 24-hydrolase in therapy of Huntington's disease
View Patent ↗The present invention relates to a vector for use in the treatment of Huntington's disease, which vector comprises a cholesterol 24-hydroxylase encoding nucleic acid.
1. A method of treating Huntington's disease in a subject in need thereof, comprising
administering to said subject a vector comprising a nucleic acid sequence that encodes cholesterol 24-hydroxylase.
2. The method of claim 1 , wherein said nucleic acid sequence encodes the amino acid sequence set forth in SEQ ID NO: 2.
3. The method of claim 1 , wherein said nucleic acid sequence comprises the nucleic acid sequence set forth in SEQ ID NO: 1.
4. The method of claim 1 , wherein said vector is selected from the group consisting of an adenovirus vector, a retrovirus vector, a herpes virus vector and an Adeno-Associated Virus (AAV) vector.
5. The method of claim 4 , wherein said vector is an AAV vector.
6. The method of claim 5 , wherein said vector is an AAV vector selected from the group consisting of AAV1, AAV2, AAV3, AAV4, AA5, AAV6, AAV7, AAV8, AAV9, and AAV10.
7. The method of claim 6 , wherein said AAV vector is an AAV10 vector.
8. The method of claim 1 , wherein said vector is administered directly into the brain of the subject.
9. The method of claim 8 , wherein said vector is administered by stereotaxic microinjection.
10. The method of claim 9 , wherein said vector is administered to the ruber nucleus, corpus amygdaloideum, entorhinal cortex and neurons in ventralis lateralis, or to the anterior nuclei of the thalamus.
11. The method of claim 1 , wherein said vector is administered by intravenous injection.
12. The method of claim 1 , wherein said vector is administered by intrathecal injection.
13. The method of claim 1 , wherein said vector is administered into the ventricles.