Abstract
During the past twenty years, gene editing has emerged as a novel form of gene therapy. Since the publication of the first potentially therapeutic gene editing platform for genetic disorders, increasingly sophisticated editing technologies have been developed. As with viral vector mediated gene addition, inborn errors of immunity (IEIs) are excellent candidate diseases for a corrective autologous haematopoietic stem cell gene editing strategy. Research on gene editing for IEIs is still entirely preclinical, with no trials yet underway. However, with editing techniques maturing, scientists are investigating this novel form of gene therapy in context of an increasing number of IEIs. Here, we present an overview of these studies and the recent progress moving these technologies closer to clinical benefit.
Published Version
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