Method of treating neurodegenerative disease
View Patent ↗Aspects featured in the invention relate to compositions and methods for inhibiting alpha-synuclein (SNCA) gene expression, such as for the treatment of neurodegenerative disorders. An anti-SNCA agent featured herein that targets the SNCA gene can have been modified to alter distribution in favor of neural cells.
1. A method of inhibiting alpha-synuclein (SNCA) expression in a mammal comprising administering an iRNA agent directly to the brain of said mammal, wherein the iRNA agent comprises an antisense strand comprising SEQ ID NO: 22 and a sense strand comprising SEQ ID NO: 21, wherein each strand is 21-25 nucleotides in length.
2. The method of claim 1 , further comprising a step of identifying a synucleinopathy in said mammal prior to the administering of said iRNA agent.
3. The method of claim 1 , wherein said SNCA expression is reduced by at least 50% relative to expression in cells that had not been contacted by said iRNA agent.
4. The method of claim 2 , wherein said mammal is a human.
5. The method of claim 1 , wherein said iRNA agent is administered by stereotactic injection into the brain of said mammal.
6. The method of claim 1 , wherein said iRNA agent is administered by intraparenchymal infusion or injection.
7. The method of claim 4 , wherein said iRNA agent is administered by stereotactic injection into the brain of said human.
8. The method of claim 4 , wherein said iRNA agent is administered by intraparenchymal infusion or injection.
9. The method of claim 1 or 4 , further comprising a step of measuring SNCA expression in said mammal.
10. The method of claim 1 or 4 , wherein said SNCA expression is measured before and after said administration of said iRNA agent.
11. The method of claim 1 , wherein each strand of the iRNA agent is 21 nucleotides in length.