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For his exact mutation, if you think about DNA, you have two copies of everything. He has one good copy and one abnormal copy. Not only is his bad copy improperly functioning, it’s also preventing the healthy copy from functioning properly, a knockdown ASO would target the abnormal copy of the gene, by preventing it from turning into the abnormal protein, without affecting the healthy copy.
While this is about Henry, it is also about many other patients whose lives can be impacted by this therapy.
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