ADMINISTRATION OF TAU REPRESSORS VIA THE PENETRATING CAPSID OF THE BLOOD-BRAIN BARRIER
Patent Information
- Application Number
- ARP20250100678
- Authority / Receiving Office
- AR · AR
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2024-11-12
- Filing Date
- 2025-03-13
- Publication Date
- 2026-09-16
Claims
Compositions are provided for the delivery of zinc finger fusion proteins that inhibit tau expression in the nervous system using blood-brain barrier penetrating AAV capsid proteins comprising SEQ ID No. 1235, and methods for the use of the compositions to treat neurodegenerative diseases such as Alzheimer's disease, frontotemporal dementia, and other tauopathies.
1. A composition comprising: 1) an adeno-associated virus (AAV) capsid protein comprising an amino acid sequence, wherein the amino acid sequence comprises at least 3, 4, 5, 6, 7, 8 or 9 contiguous amino acids from the amino acid sequence established in SEQ ID No. 71; and 2) an expression construct comprising a coding sequence for a fusion protein, wherein the fusion protein comprises a zinc finger protein (ZFP) domain and a transcription repressor domain, wherein the ZFP domain binds to a target region of a human microtubule-associated tau protein (MAPT) gene.
31. A nucleic acid construct encoding the expression construct of any of claims 1 or 8 - 21.
32. A pharmaceutical composition comprising the composition of any of claims 1-21 or the nucleic acid construct of claim 31, and a pharmaceutically acceptable carrier.