SARM1 Antisense Oligonucleotides for Axonal Degeneration Prevention
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Solution Overview
Problem
Axonal degeneration is a hallmark of neurological disorders and neurodegenerative diseases, causing significant devastation and high costs, with existing treatments lacking effective solutions to prevent or slow this process.
Innovation Solution
The use of antisense oligonucleotides targeting Sterile Alpha and TIR motif-containing 1 (SARM1) to inhibit its function, thereby preventing axonal degeneration, is proposed. These oligonucleotides include sequences with specific identities and modifications, administered to subjects to decrease SARM1 mRNA and protein levels.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If existing treatments are used for neurological disorders, then current therapeutic options are limited, but axonal degeneration continues to progress causing devastation and high costs
Solution Approach 1:
The patent extracts and targets the specific molecular culprit SARM1 from the complex axonal degeneration pathway. By designing antisense oligonucleotides that specifically bind to SARM1 mRNA, the invention isolates and neutralizes the harmful executioner molecule responsible for axonal degeneration, thereby preventing neurodegeneration without affecting other cellular processes
Solution Approach 2:
The patent introduces antisense oligonucleotides as intermediary molecules that mediate between the administered therapy and the SARM1 target. These oligonucleotides serve as the intermediate mechanism that delivers the therapeutic effect by binding to SARM1 mRNA and preventing its translation, thus acting as a bridge between external treatment and internal pathological process
2Object-affected harmful factors
If antisense oligonucleotides targeting SARM1 are administered, then SARM1 levels are reduced and axonal degeneration is prevented, but the complexity of the treatment increases
Solution Approach 1:
The patent replaces complex mechanical or surgical interventions with a molecular-level biochemical mechanism. Instead of invasive procedures or complex device-based treatments, the invention uses antisense oligonucleotides that exploit natural cellular processes (mRNA degradation pathways) to achieve therapeutic effects, thereby simplifying the overall treatment approach while maintaining high efficacy
Applied Scientific Principles
This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.
Function Achieved in This Case
The antisense oligonucleotides effectively reduce SARM1 levels, potentially preventing or slowing axonal degeneration in various neurological disorders, including peripheral and central nervous system diseases, thereby reducing disease progression and associated costs.
Implementation Method 1
antisense oligonucleotides that inhibit SARM1 are particularly beneficial for preventing axonal degeneration
Implementation Method 2
antisense oligonucleotides comprising a sequence having at least 80% identity to a sequence selected from a group consisting of SEQ ID NO: 3-21, 23-26, 38 and 39
Data Source
AI summary
The present invention relates to compositions and methods for treating neurodegeneration and neurodegenerative diseases associated with axonal degeneration. Neurodegeneration and neurodegenerative diseases associated with axonal degeneration are treated with therapies comprising SARM1 inhibitors such as SARM1 antisense oligonucleotides.