CRISPR—Deliverer or Disruptor?

Inborn errors of metabolism (IEM) cause hereditary multisystemic metabolic diseases (HMD), and classically they result from one or more dysfunctional enzymes or defects in the transportation of proteins. The dysfunctionality is caused by mutations in genes that code for the enzymes; enzyme deficiency or inactivity leads to accumulation of substrate precursors or metabolites, or to frank deficiencies of the enzyme’s products (Camp 2012) (Scriver, Sly, Childs, et al. 2000).

IEM are not rare …