Table 2.

An overview of typical applications of gene augmentation and genome editing technologies.

Application of Gene Augmentation	Application of Genome Editing
To restore functional protein expression for loss-of-function mutations	To correct loss-of-function and knock-out toxic-gain-of-function mutations
To produce enzymes in the liver that upon secretion are delivered to other tissues, i.e., lysosomal enzymes	To correct mutations in genes that need tight control by their endogenous promoter
	To modify a metabolic process, i.e., knocking out PCSK9 expression to lower serum cholesterol