CRISPR/Cas9

CRISPR/Cas9 – a specific, efficient and versatile gene-editing technology we can harness to modify, delete or correct precise regions of our DNA

Dr. Emmanuelle Charpentier, one of our scientific founders, co-invented CRISPR/Cas9 gene editing. Until then, people knew “CRISPR” only as an acronym for the Clustered Regularly Interspaced Short Palindromic Repeats of genetic information that some bacterial species use as part of an antiviral mechanism. Now, as a gene-editing tool, CRISPR/Cas9 has revolutionized biomedical research and may soon enable medical breakthroughs in a way few biological innovations have before.

CRISPR/Cas9 edits genes by precisely cutting DNA and then letting natural DNA repair processes to take over. The system consists of two parts: the Cas9 enzyme and a guide RNA.

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Rapidly translating a revolutionary technology into transformative therapies.

Rapidly translating a revolutionary technology into transformative therapies.


CRISPR Lexicon

  • CRISPR: Clustered Regularly Interspaced Short Palindromic Repeats of genetic information that some bacterial species use as part of an antiviral system. A group of scientists, including our co-founder Dr. Emmanuelle Charpentier, discovered how to use this system as a gene-editing tool (Jinek, et al. Science 2012)
  • Cas9: a CRISPR-associated (Cas) endonuclease, or enzyme, that acts as “molecular scissors” to cut DNA at a location specified by a guide RNA
  • Deoxyribonucleic acid (DNA): the molecule that most organisms use to store genetic information, which contains the “instructions for life”
  • Ribonucleic acid (RNA): a molecule related to DNA that living things use for a number of purposes, including transporting and reading the DNA “instructions”
  • Guide RNA (gRNA): a type of RNA molecule that binds to Cas9 and specifies, based on the sequence of the gRNA, the location at which Cas9 will cut DNA

Three main categories of genetic edits can be performed with CRISPR/Cas9:

CRISPR/Cas9 Gene Editing

Disruption2

DISRUPT

If a single cut is made, a process called non-homologous end joining can result in the addition or deletion of base pairs, disrupting the original DNA sequence and causing gene inactivation

Deletion

DELETE

A larger fragment of DNA can be deleted by using two guide RNAs that target separate sites. After cleavage at each site, non-homologous end joining unites the separate ends, deleting the intervening sequence

Correction

CORRECT OR INSERT

Adding a DNA template alongside the CRISPR/Cas9 machinery allows the cell to correct a gene, or even insert a new gene, using a process called homology directed repair

"If scientists can dream of a genetic manipulation, CRISPR can now make it happen"


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Our CTX001 program in hemoglobinopathies uses disruption to achieve the targeted gene edit

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Our CTX110 CAR-T program uses disruption and insertion to create the desired allogeneic CAR-T cell

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