CRISPR Health Applications
“[CRISPR is] a tool that scientists and clinicians around the world are using to understand our genetics, the genetics of all living things, and — most importantly — to intervene in genetic disease.”
Jennifer Doudna, PhD, University of California, Berkeley
What is CRISPR?
1993
2005
2008
2010
2010 - 2011
2012
2013
2018
2020
Haloferax mediterranei: an archaeal microbe
Francisco Mojica, a researcher from the University of Alicante found the unknown “frequent repetitive motifs” in the Haloferax mediterranei DNA fragment during his Ph.D. study.
Dr. Mojica termed these short repeated DNA sequences “Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR),” characterized by the regular spacing between the recurring units with similar sequences of sizes. He hypothesized the connection between CRISPR loci and the adaptive immune system against infections.
​
Read about "Intervening Sequences of Regularly Spaced Prokaryotic Repeats Derive from Foreign Genetic Elements"
Dr. Marraffini and Dr. Sontheimer confirmed that the target of CRISPR interference was DNA and envisioned this programmable and specific restriction enzyme for future gene editing in clinical applications.
​
Read about "CRISPR Interference Limits Horizontal Gene Transfer in Staphylococci by Targeting DNA"
Dr. Moineau and his colleagues demonstrated ​​site-specific double-stranded breaks in DNA guided by Cas9 nuclease. The targeted position was encoded by short palindromic repeated RNA (crRNAs).
​
Trans-activating crRNA (tracrRNA) was characterized to be involved in crRNA maturation and DNA cleavage by the Cas9 nuclease complex.
​
Read about "[the] trans-encoded small RNA [that] directs processing of precursor RNA into crRNAs through endogenous RNase III and the CRISPR-associated Csn1 protein"
Dr. Emmanuelle Charpentier and Dr. Jennifer Doudna used recombinant Cas9, crRNA, and tracrRNA to cut DNA in vitro. The two-RNA structure consisting of custom-designable crRNA and its pairing tracrRNA guided Cas9 to introduce targeted double-stranded breaks, and this again shows the possibility of future RNA-programmable genome editing.
Dr. Feng Zhang and Dr. George Church's group individually reported engineered short RNA-guided site-specific DNA cleavage from the type II prokaryotic clustered regularly interspaced short palindromic repeats (CRISPR) adaptive immune system as a genome editing tool that is achievable in human and mouse cells.
​
Read about Multiplex Genome Engineering Using CRISPR/Cas System
​
Read about RNA-Guided Human Genome Engineering via Cas9
​
​
​
The CRISPR-baby scandal
Despite this exciting tool for "genome surgery," the fast-moving field also raises ethical concerns. A Chinese scientist, He Jiankui, announced the first gene-edited twin girls with a modified CCR5 gene. The goal was to mitigate the father-to-child HIV transmission and to "produce an [in vitro fertilization] baby naturally immunized against AIDS".
​
Perspective: Germline editing - Crossing ethical red lines in gene editing
​
Nobel Prize in Chemistry: The Age of CRISPR
Jennifer Doudna and Emmanuelle Charpentier have been awarded the 2020 Nobel Prize in Chemistry due to their key contribution in discovering the revolutionary genome editing tool. CRISPR technology is not only commonly used in research labs, but it also has a significant impact on advancing clinical therapeutics and potential in translational medicine.
Get a detailed overview of the CRISPR timeline
