CRISPR

CRISPR is the acronym for "clustered regularly interspaced short palindromic repeats".

Advances in DNA sequencing make it possible to identify the genetic triggers of diseases. It is now possible to edit genomic DNA sequences precisely and with high efficiency using "CRISPR/Cas9 technology". For example, bacteria and archaea use repetitive parts of the genome (CRISPR), in combination with an RNA-dependent nuclease(CRISPR-associated nuclease=Cas), to recognize and destroy DNA that invades cells. By appropriately choosing the sequence of the guide RNA (guide RNA), this dual system can be used to efficiently target or modify gene loci in plants, insects, fungi, mammalian cells, and whole organisms. This opens up unimagined possibilities in genetic engineering, up to and including the treatment or cure of human hereditary diseases.

A first attempt to cure a genetic disease using CRISPR/Cas9 technology is currently being made by the biotech company Verve Therapeutics (New Zealand) in heterozygous familial hypercholesterolemia. The drug is designed to permanently switch off the gene for the proportein convertase subtilisin/kexin type 9 (PCSK9) in the liver in order to lower the disease-promoting low-density lipoprotein-cholesterol (LDL-C).

1. Kleinstiver BP et aal. (2015) Engineered CRISPR-Cas9 nucleases with altered PAM specificities. Nature 523:481-485
2. Oppel Fet al. (2019) Perspectives on genome editing in otolaryngology. HNO 67: 184-189