Epigenetic editors at ASGCT: animal models address the durability question
As the new modality advances, the promise of durability is bearing out in preclinical models
Gene editing is attractive partly because of its one-and-done capability, but there are safety risks to cutting the DNA and permanently altering its sequence. Modifying the epigenome promises a more attractive safety profile, but the first step is for companies to show the resulting changes in gene expression are sufficiently long-lasting for therapeutic use.
Data in abstracts from ASGCT hint that epigenome modifications, which have already proven heritable during cell replication in vitro, can lead to sustained changes in protein expression in animals...
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