Cardiovascular diseases (CVD) are the major cause of death in the USA (pre-COVID) and became the top risk factor for causing mortality after COVID19 viral infection. CVDs are rising owing to increasingly sedentary lifestyles and unhealthy diets. Additionally, the rising geriatric population has further increased the incidences of cardiovascular diseases, globally. A number of novel therapeutics have been launched in the global market and have been highly successful, but the nature of CVD renders it almost incurable and the rising prevalence is further worsening the treatment outlook for patients. This has led to the development of a huge unmet treatment need in the market.
A protein known as transcription factor GATA-binding protein 4 (GATA4) is a major regulator of cardiac enlargement or hypertrophy, a major negative outcome from most CVDs that reduces cardiac function. Cells utilize messenger ribonucleic acid (mRNA) as a blueprint for protein sequence and this resulting protein will then participate in cellular processes. Some diseases manifest from changed abundances of these proteins, like GATA4, and Antisense Oligonucleotides (ASOs) are often used to downregulate the gene expression.
Researchers at the University of Rochester have developed a set of ASOs (GATA4-ASO) for targeting GATA4 mRNA. Using this technology, they have treated cardiomyocytes (cardiac muscle cells) in vitro and observed a significant reduction of GATA4 protein expression and repressed cardiomyocyte hypertrophy. In two pre-clinical trials, GATA4-ASO reduced GATA4 protein expression, thereby antagonizing cardiac hypertrophy and restoring normal cardiac function as indicated by echocardiography. Results indicate that this GATA4 mRNA is a potential anti-hypertrophic therapeutic target and RNA-based ASO may be considered a potential pharmaceutical drug candidate to treat heart disease.
Pre-clinical success in the form of reduced cardiac enlargement and enhanced cardiac function in animal heart failure disease models. This ASO system can be used to enhance the translation of protective and beneficial proteins, while also repressing the translation of the pathogenic and detrimental proteins in any disease conditions.
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- Provisional Patent Application Filed
- Provisional patent
- Patent application submitted
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Name: Matan Rapoport