Silencing the genes that harm the brain

摘要

Instead of trying to mop up diseasecausing proteins once they have been produced, like most small molecule or antibody therapies, genesilencing treatments target messenger RNA (mRNA) to cut off production of these proteins at source. And pioneering pharmaceutical companies working on this technology now have neurodegenerative diseases in their sights. Neurodegenerative diseases such as Alzheimer's, Parkinson's and Huntington's -are common and most lack diseasemodifying therapies or cures.