RNAi-Based Treatment for Chronic Liver Diseases

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According to the 2010 National Hospital Discharge Survey as published by the CDC, over 101,000 patients in the United States were hospitalized due to chronic liver disease, including fatty liver disease, cirrhosis, alcoholic liver disease, and liver cancer. In the same year, there were 36,427 deaths related to chronic liver disease. One of the strongest genetic risk factors for developing chronic liver disease is the I148M mutation of the PNPLA3 gene. In models, it was confirmed that reducing the 148M isoform resulted in reduced fat accumulation, which affects the development of fatty liver disease in humans. Reducing the expression of this 148M isoform is a promising mechanism for slowing or reversing the progression of chronic liver disease.

Researchers at Purdue University have developed a small-interfering RNA (siRNA) that specifically targets the 148M isoform. This siRNA reduces the expression of 148M mRNA while having minimal effect on the 148I isoform. This siRNA shows promise as a therapy for fatty liver disease and other types of chronic liver disease.

-Targets the genetic mutation that causes chronic liver disease
-Affects the responsible isoform with minimal other effects
-Models have shown reduced fat accumulation

Potential applications:
-Therapy for chronic liver disease
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