rAAV Vector Encoding NPY and NPY2R for Pharmacoresistant Epilepsy
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Solution Overview
Problem
Current therapeutic options for neurological disorders such as epilepsy are insufficient, particularly for pharmacoresistant cases, and there is a need for new approaches to effectively treat these conditions.
Innovation Solution
A recombinant adeno-associated viral (rAAV) vector encoding neuropeptide Y (NPY) and its receptor Y2 (NPY2R) is developed, encapsulated in adeno-associated virus (AAV) capsid proteins, and used to deliver a pharmaceutical composition for treating neurological disorders like epilepsy and Parkinson's disease by administering it to the central nervous system.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If current therapeutic options are used for neurological disorders, then treatment is available for some cases, but treatment is insufficient for pharmacoresistant cases
Solution Approach 1:
The invention segments the therapeutic approach by delivering separate coding sequences for NPY and NPY2R receptors via AAV vector, allowing independent expression and action of each component to address pharmacoresistant epilepsy through coordinated neuropeptide-receptor interaction
Solution Approach 2:
The AAV vector serves as an intermediary delivery system that introduces exogenous NPY and NPY2R coding sequences into the central nervous system, enabling therapeutic expression in pharmacoresistant cases where conventional treatments fail
2Reliability
If neuropeptide Y is overexpressed alone, then anti-seizure effects are achieved, but receptor downregulation limits therapeutic effect over time
Solution Approach 1:
The invention merges the NPY coding sequence with NPY2R receptor coding sequence in a single AAV vector, ensuring co-expression of both the neuropeptide and its specific receptor, thereby preventing receptor downregulation and maintaining sustained therapeutic effect
Solution Approach 2:
The co-expression of NPY2R receptors provides a feedback mechanism that prevents compensatory downregulation, maintaining stable NPY signaling pathways and sustained anti-seizure therapeutic effect over time
3Duration of action of stationary object
If AAV vector encodes both NPY and NPY2R, then sustained therapeutic effect is achieved, but vector complexity increases
Solution Approach 1:
The AAV vector is designed with multi-functionality, serving as a single delivery vehicle that simultaneously introduces both NPY and NPY2R coding sequences, eliminating the need for separate vectors and simplifying the overall therapeutic system
Data Source
AI summary
The present invention relates to a recombinant adeno-associated viral (r AAV) vector comprising neuropeptide Y (NPY) coding sequence and neuropeptide Y2 receptor (NPY2R) coding sequence. The invention further relates to a AAV particle comprising said vector, wherein the vector is encapsulated by adeno-associated virus (AAV) capsid proteins. Also, a pharmaceutical composition comprising said AAV particle, for use in the prevention or treatment of a neurological disorder in mammals, such as epilepsy.