Dual Gene Construct SERCA2a CCN5 Heart Failure Treatment
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Solution Overview
Problem
Current treatments for heart failure primarily focus on relieving symptoms and slowing disease progression, but they do not address the underlying pathological cardiac remodeling and cardiomyocyte damage, leading to a high mortality rate and significant treatment costs.
Innovation Solution
A gene construct comprising nucleotide sequences encoding SERCA2a and CCN5 proteins, which are used to create a recombinant expression vector or virus, is administered to treat heart failure. This combination aims to prevent cardiomyocyte loss and fibrosis, thereby addressing the complex nature of heart failure.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If current treatments for heart failure are used, then symptom relief and disease progression slowing is achieved, but underlying pathological cardiac remodeling and cardiomyocyte damage are not addressed, leading to high mortality rate
Solution Approach 1:
The patent combines two gene therapy approaches (SERCA2a overexpression and CCN5 suppression) into a single comprehensive treatment strategy. The dual-target approach addresses both cardiomyocyte loss and fibrosis simultaneously, aiming to reverse pathological remodeling while improving cardiac function, thereby tackling the root causes rather than just symptoms
2Productivity
If current treatments are used, then symptom management is provided, but treatment costs become a significant burden on patients
Solution Approach 1:
The patent replaces conventional pharmacological treatments with gene therapy technology. By using viral vectors to deliver therapeutic genes and suppress harmful genes, the treatment aims to achieve durable effects through biological mechanisms rather than continuous drug administration, potentially reducing long-term treatment costs despite high initial investment
3Reliability
If SERCA2a and CCN5 proteins are simultaneously expressed, then synergistic therapeutic effect is achieved by preventing cardiomyocyte loss and fibrosis, but the complexity of the gene construct increases
Solution Approach 1:
The patent merges two separate genetic interventions into a coordinated system. The SERCA2a gene is delivered via viral vector for overexpression, while CCN5 is suppressed using RNA interference mechanisms. This combined approach targets both the loss of functional cardiomyocytes and the pathological fibrosis, creating a synergistic effect that addresses multiple aspects of heart failure pathophysiology simultaneously
Solution Approach 2:
The gene construct employs composite genetic elements including promoter regions, coding sequences, and regulatory elements that work together to achieve controlled expression of SERCA2a and suppression of CCN5. The use of viral vectors combined with RNA interference mechanisms creates a multi-component therapeutic system that leverages different biological pathways for enhanced efficacy
Data Source
AI summary
The present invention relates to a pharmaceutical composition for prevention and treatment of heart failure. Specifically, the present invention relates to a gene construct comprising a polynucleotide coding for SERCA2a protein or a fragment thereof and a polynucleotide coding for CCN5 protein or a fragment thereof, and a pharmaceutical composition comprising the same construct as an effective ingredient for preventing or treating heart failure. A pharmaceutical composition for prevention and treatment of heart failure according to the present invention is used in a method for co-expression of SERCA2a protein and CCN5 protein. Designed to exert a synergistic therapeutic effect through SERCA2a protein's function of preventing the loss of cardiomyocytes and increasing the activity of cardiomyocytes and CCN5 protein's function of suppressing the fibrosis of heart cells and tissues, the pharmaceutical composition can be useful for preventing or treating heart failure, which is a complex disorder induced by various etiological factors.


