Key result
Research on gene editing for inborn errors of immunity remains entirely preclinical, with no clinical trials yet underway, though techniques are maturing toward clinical application.
Why the study?
Gene editing technologies are maturing and being investigated across an increasing number of inborn errors of immunity, moving preclinical autologous hematopoietic stem cell gene editing closer to clinical benefit.
Gene editing represents a promising, though currently preclinical, therapeutic strategy for inborn errors of immunity.
Should not yet change practice for inborn errors of immunity; leaves open clinical translation as techniques mature.
During the past 20 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 are excellent candidate diseases for a corrective autologous hematopoietic stem cell gene editing strategy. Research on gene editing for inborn errors of immunity 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 inborn errors of immunity. Here, we present an overview of these studies and the recent progress moving these technologies closer to clinical benefit.
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Mudde et al. (2024) conducted a review in Inborn errors of immunity. Genome editing was evaluated. Research on gene editing for inborn errors of immunity remains entirely preclinical, with no clinical trials yet underway, though techniques are maturing toward clinical application.
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