CRISPR-Cas9 based gene editing strategies for the correction of genetic disorders Congenital genetic defects are the leading cause of morbidity and mortality. Despite advances in our understanding of the etiology and pathophysiology of genetic diseases, treatments are lacking. Advances in precision medicine, specifically gene therapy and editing approaches, have provided hope for a cure for these diseases at their molecular roots and can help in improving pathological outcomes. Based on the route of administration, time of delivery (prenatal or postnatal) and type of vectors (viral vectors and nanoparticles), different cell types and tissues can be targeted. In this talk, I will discuss nanoparticle-mediated gene editing (including base editing) strategies for the treatment of diseases such as type 1 tyrosinemia.
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April 2024
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