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Gene and gene-modified cell therapy treatments have demonstrated their potential in addressing unmet medical needs across a wide variety of human diseases, including cardiovascular, neurodegenerative, ocular, immunologic disorders and cancer. These therapies are based on the delivery of corrective DNA (DeoxyriboNucleic Acid) into target cells with viral vectors.
Over the past three decades, research has been focused on addressing the unmet needs of drug-based treatments by developing gene-based therapy. Based on genome modification through intracellular delivery of engineered exogenous nucleic acids, gene-based therapy is extensively investigated for its use as a stand-alone treatment or in combination with existing drug treatments. Gene-based therapies are generally divided into two categories: viral and non-viral vector mediated therapies.
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