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A Breakthrough in Genetic Medicine for Rare Diseases
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A Breakthrough in Genetic Medicine for Rare Diseases

A long-disdained therapy that targets RNA is suddenly achieving spectacular success By Lydia Denworth | Scientific American March 2020 Issue Antisense oligonucleotides (ASOs) are short strings of chemically modified DNA or RNA that are designed to home in on RNA strands to alter the proteins the body ultimately produces. After decades of struggling to get...