One of the major advantages of in situ gene correction strategies is the possibility of restoring both the function and the expression control of the affected gene (Ferrari et al., Frontiers in Genome Editing 2021). This issue becomes even more relevant when the affected gene is directly active in cell differentiation and proliferation because its ectopic or constitutive expression may trigger uncontrolled cell expansion with a significant risk of oncogenic transformation, such as in the case of the CD40LG gene in the X-linked hyper-IgM syndrome (HIGM1).
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