“DNA is like a computer program but far, far more advanced than any software ever created.”
― Bill Gates
In 2020, Drs. Jennifer Doudna and Emmanuelle Charpentier won the Nobel Prize in Chemistry for developing the gene-editing tool known as CRISPR. In the words of the Nobel committee that honored Drs. Charpentier and Doudna, CRISPR has "reshaped the life sciences," with almost endless examples of how CRISPR-Cas9 could be used …"
The promise recognized by the Nobel Committee just five years ago is now bearing fruit, and the rate of harvest appears to be accelerating. On March 24, 2025, Verve Therapeutic (VERV) announced regulatory approval for clinical trials of its CRISPR-based treatment for heterozygous familial hypercholesterolemia (HeFH), an inherited condition that causes elevated blood concentration of low-density lipoprotein (LDL) cholesterol (aka "bad" cholesterol) and is associated with premature coronary artery disease. The company hopes tests will show that a single dose of its VERVE-102 treatment can turn off the gene that causes HeFH.
Charpentier and Doudna's discovery built on decades of research into a defense mechanism used by the Streptococcus pyogenes bacteria to fight off viral infections. In essence, the bacteria use RNA to copy and record snippets of the genetic codes of viruses that they encounter. When re-exposed to one of those viruses, the bacteria can use this guide RNA to quickly recognize it and bind a DNA-slicing enzyme – a protein known as Cas9, to those genetic snippets – thus severing the viruses' genetic code and rendering them unable to replicate.
CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) mimics that process, creating "guide RNAs" to target specific gene sequences on a DNA double-helix and bind Cas proteins to them. If the Cas protein used is Cas9, this essentially allows scientists to snip our genes with precision, and either nullify or replace them. The CRISPR process is superior to previous methods of gene editing in that it eliminates the need to laboriously and slowly engineer a new, unique protein for each gene to be targeted.
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