Epigenome-Modifying Agents Prevent Protein Degradation
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Solution Overview
Problem
Current treatments for muscular genetic diseases caused by protein conformation defects, such as sarcoglycanopathies and dystrophies, are ineffective in preventing the degradation of abnormal proteins, leading to progressive muscle degeneration and loss of locomotor functions.
Innovation Solution
A pharmaceutical composition comprising epigenome-modifying agents, including DNA methylation inhibitors and histone deacetylase inhibitors, is used to treat muscle genetic diseases by preventing the cellular degradation of proteins with conformational defects, thereby restoring their functionality.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If conventional treatments are used for muscular genetic diseases, then current therapeutic options are limited, but the abnormal proteins are degraded by cellular quality control systems
Solution Approach 1:
The invention changes the biochemical parameters of the cell by using HDAC inhibitors to modify histone deacetylation, which alters the epigenetic state and prevents the degradation of misfolded proteins. This parameter change in chromatin structure allows the abnormal proteins to be retained and potentially refolded, addressing both protein stability and treatment versatility
Solution Approach 2:
The invention introduces HDAC inhibitors as intermediary substances that mediate between the cellular quality control system and the misfolded proteins. These compounds act as intermediaries that modify the epigenetic environment, thereby interfering with the degradation pathway and allowing the abnormal proteins to persist and potentially regain function
2Reliability
If the abnormal protein is allowed to accumulate, then protein functionality may be restored, but the cellular quality control system degrades the misfolded protein
Solution Approach 1:
The invention converts the harmful degradation process into a beneficial outcome by using HDAC inhibitors to prevent degradation. The cellular quality control system's degradation pathway, which normally eliminates misfolded proteins, is epigenetically modulated to retain these proteins, allowing them to accumulate and potentially refold into functional conformations
Solution Approach 2:
The invention applies preliminary anti-action by using HDAC inhibitors to preemptively block the degradation pathway before misfolded proteins are destroyed. By modifying the epigenetic state in advance, the cell is prepared to retain abnormal proteins rather than degrade them, preventing the loss of potentially functional protein variants
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 use of epigenome-modifying agents effectively prevents the degradation of misfolded proteins, allowing them to localize correctly in muscle cells and restore normal muscle function, offering a new therapeutic approach for progressive muscular dystrophies.
Implementation Method 1
a DNA methylation inhibitor, advantageously a methyltransferase inhibitor
Implementation Method 2
a histone deacetylase inhibitor
Data Source
Figure 1A~2C
AI summary
The invention relates to a pharmaceutical composition including at least one epigenome-modifying compound, for the use thereof in the treatment of genetic muscular diseases linked to a conformational anomaly of at least one protein, said anomaly leading to the cellular degradation of the protein.