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Bree holds both a BSc and PhD in Genetics from the University of Liverpool. After completing her studies, she spent two years as a science writer at an agency. Eager to broaden her expertise, she joined Technology Networks as a science writer in 2024. In her current role, she is responsible for producing custom written content and contributing to the development of digital media.
From a bacterial immune defense system to a versatile gene-editing tool, CRISPR technology has transformed genetic research by enabling precise modifications in living cells.
Scientists are continuing to refine CRISPR techniques to enhance precision, reduce off-target effects and expand its applications beyond DNA editing.
This infographic explores the history, breakthroughs and future directions of CRISPR-Cas systems, highlighting their transformative role in gene editing and personalized medicine.
Download this infographic to explore:
Key milestones in CRISPR’s evolution and its expanding applications
How advanced editing techniques are refining precision and efficiency
The future of CRISPR-based therapies and their clinical potential
CRISPR
Technology:
How Far Have
We Come?
Genome editing involves modifying DNA at
specific target sites, through insertion, deletion
or substitution. These changes are typically
made to inactivate genes or introduce novel
gene sequences in living cells.1
What is CRISPR?
CRISPR, short for Clustered Regularly
Interspaced Short Palindromic Repeats, is
a natural defense system found in many
bacteria and archaea.2 These systems
capture and store fragments of foreign DNA,
such as those from phages, in repetitive
arrays. These stored DNA sequences then
serve as molecular guides for CRISPRassociated
(Cas) proteins, which precisely
target and destroy invading genetic material.
CRISPR-Cas systems are at the forefront of gene editing
techniques, due to their advantages of simple design,
robust activity and their capacity to target virtually any
DNA or RNA site.
This infographic provides an in-depth look at CRISPR-Cas
systems, their history, advancements and transformative
impact on gene editing and personalized medicine.
DNA insertion DNA deletion DNA substitution
Immunization
Immunity
A guide RNA (gRNA) is
created to match the DNA
sequence of the target.
This activates the cell’s natural repair mechanisms, resulting in
small insertions or deletions that disrupt the gene sequence or the
integration of a new gene sequence.
The gRNA directs Cas9 to the specific target sequence in the DNA,
enabling Cas9 to create a double-strand break at the targeted location.
This is paired with the Cas9
protein and delivered into the
target organism.
Scientists have adapted this system to edit DNA in plants, animals and humans with remarkable precision.
The process works as follows:
1987
The CRISPR mechanism is first
discovered in Escherichia coli.3
2002
Scientists discover more clustered
repeats of DNA in bacteria and
archaea, and the term CRISPR-Cas9
is coined.4, 5
2012
Emmanuelle Charpentier, Jennifer
Doudna and colleagues publish their
landmark paper in Science on CRISPRCas9
as a genome-editing tool.2
2015
Cas 12 and 13 are discovered,
expanding the CRISPR toolbox beyond
traditional genome editing, including
viral detection and programmable RNA
manipulation.6, 7
2016
The first CRISPR-based therapy is
used to treat a patient with lung
cancer in a clinical trial conducted at
West China Hospital in Sichuan, China.8
2018
Biophysicist He Jiankui announces the
creation of CRISPR-edited babies who
are immune to HIV, sparking global
ethical debates.9
2020
Charpentier and Doudna are awarded
the Nobel Prize in Chemistry for their
work on CRISPR.10
2023
World’s first CRISPR–Cas9 gene
editing therapy receives conditional
approval in the UK for the treatment of
two blood disorders.11
2024
An in vivo CRISPR gene therapy clinical
trial effectively targets and repairs a
genetic mutation responsible for a rare
type of blindness.12
CRISPR timeline: A genetic revolution
The next generation of CRISPR systems
CRISPR-Cas technology has evolved far beyond its origins as a DNA-cleaving tool. Today, it encompasses
a diverse suite of techniques, including single-base gene editing, transcriptional regulation and RNA
strand modification.
Cas9 nickase: Editing with single-strand breaks
Cas9 nickase is a modified version of the Cas9
protein that introduces single-strand breaks (nicks)
in DNA instead of double-strand breaks (DSBs). This
modification reduces the risk of off-target effects and
unwanted genomic instability.
RNA Editing System: Modifying RNA instead of DNA
The CRISPR-Cas13 system provides a powerful tool for
RNA editing, enabling gene silencing without affecting
the genome. Cas13 targets and cleaves RNA, offering new
opportunities for developing nucleic acid therapeutics
and avoiding potential genomic instability.14
Dead-Cas9 System: Targeting without cutting DNA
The dead-Cas9 (dCas9) variant binds to targeted DNA
using guide RNA, but cannot cleave it.13 dCas9 can be
used to deliver virtually any cargo to specific loci in the
genome, including transcriptional activators or inhibitors,
enabling precise control of genetic activity.
Prime Editing System: Editing without DSBs
CRISPR prime editing combines a Cas9 nickase with an engineered reverse transcriptase enzyme and a prime editing
guide RNA (pegRNA). This versatile system uses pegRNA as both a targeting and template molecule, enabling precise DNA
sequence modifications without requiring DSBs, broadening the potential applications of genome editing.
Base Editing System: Single-base precision
Base editing systems utilize dCas9 fused with nucleobase
deaminase enzymes to achieve targeted single-base changes
in DNA. This approach allows for precise point mutations
without relying on DSBs or cellular repair mechanisms, making
it particularly effective in non-dividing cells.
References:
1. Xu Y, Li Z. CRISPR-Cas systems: Overview, innovations and applications in human disease research and gene therapy. Comput Struct Biotechnol J. 2020;18:2401-2415.
doi:10.1016/j.csbj.2020.08.031
2. Jinek M, Chylinski K, Fonfara I, Hauer M, Doudna JA, Charpentier E. A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity. Science.
2012;337(6096):816-821. doi:10.1126/science.1225829
3. Ishino Y, Shinagawa H, Makino K, Amemura M, Nakata A. Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli,
and identification of the gene product. Journal of Bacteriology. 1987;169(12):5429-5433. doi:10.1128/jb.169.12.5429-5433.1987
4. Mojica FJM, Díez-Villaseñor C, Soria E, Juez G. Biological significance of a family of regularly spaced repeats in the genomes of Archaea, Bacteria and mitochondria. Molecular
Microbiology. 2000;36(1):244-246. doi:10.1046/j.1365-2958.2000.01838.x
5. Jansen Ruud, Embden JanDA van, Gaastra Wim, Schouls LeoM. Identification of genes that are associated with DNA repeats in prokaryotes. Molecular Microbiology.
2002;43(6):1565-1575. doi:10.1046/j.1365-2958.2002.02839.x
6. Zetsche B, Gootenberg JS, Abudayyeh OO, et al. Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System. Cell. 2015;163(3):759-771. doi:10.1016/j.
cell.2015.09.038
7. Shmakov S, Abudayyeh OO, Makarova KS, et al. Discovery and Functional Characterization of Diverse Class 2 CRISPR-Cas Systems. Molecular Cell. 2015;60(3):385-397.
doi:10.1016/j.molcel.2015.10.008
8. Lu Y, Xue J, Deng T, et al. Safety and feasibility of CRISPR-edited T cells in patients with refractory non-small-cell lung cancer. Nat Med. 2020;26(5):732-740. doi:10.1038/
s41591-020-0840-5
9. Raposo VL. The First Chinese Edited Babies: A Leap of Faith in Science. JBRA Assist Reprod. 2019;23(3):197-199. doi:10.5935/1518-0557.20190042
10. The Nobel Prize in Chemistry 2020. NobelPrize.org. Accessed January 21, 2025. https://www.nobelprize.org/prizes/chemistry/2020/popular-information/
11. MHRA authorises world-first gene therapy that aims to cure sickle-cell disease and transfusion-dependent β-thalassemia. GOV.UK. Accessed January 21, 2025. https://www.
gov.uk/government/news/mhra-authorises-world-first-gene-therapy-that-aims-to-cure-sickle-cell-disease-and-transfusion-dependent-thalassemia
12. Pierce EA, Aleman TS, Jayasundera KT, et al. Gene Editing for CEP290-Associated Retinal Degeneration. NEJM. Published online June 6, 2024. doi:10.1056/NEJMoa2309915
13. Brezgin S, Kostyusheva A, Kostyushev D, Chulanov V. Dead Cas Systems: Types, Principles, and Applications. Int J Mol Sci. 2019;20(23):6041. doi:10.3390/ijms20236041
14. Zhu G, Zhou X, Wen M, Qiao J, Li G, Yao Y. CRISPR–Cas13: Pioneering RNA Editing for Nucleic Acid Therapeutics. BioDesign Res. 2024;6:0041. doi:10.34133/bdr.0041
15. Macarrón Palacios A, Korus P, Wilkens BGC, Heshmatpour N, Patnaik SR. Revolutionizing in vivo therapy with CRISPR/Cas genome editing: breakthroughs, opportunities and
challenges. Front Genome Ed. 2024;6. doi:10.3389/fgeed.2024.1342193
The future of CRISPR-based therapies
With these advancements, the future of
CRISPR-Cas-mediated therapy is poised
to transform medicine, offering hope for
previously untreatable conditions and
transforming lives on a global scale.
Stored DNA sequences
cas genes
Repetitive array
Cas complex Guide RNA (gRNA)
CRISPR/Cas
Foreign DNA
L 1 2 3 4 5 6
Supressor
Off
Activator
Base editor
Base edited site
Degraded RNA
Target mRNA
gRNA
Cas13
On
Incorporation of edit into target DNA
by reverse transcriptase
Target binding and nicking Binding of primer to non-target strand
of non-target strand
Primer binding
site (PBS)
RT template
including edit
pegRNA
Scaffold
Spacer
Cas9n
Reverse transcriptase
Cellular endonucleases and mismatch repair
resolve the heteroduplex
While promising, this method is labor-intensive, costly
and challenging to scale for widespread use.
Today, most CRISPR/Cas therapies rely on ex vivo genetic
modification, where patient cells are collected, edited outside
the body and then reintroduced.
Clinical trials across the world are exploring this revolutionary
approach to address diverse conditions, from retinal disorders
to inherited blood diseases, cancers and even viral infections.15
Emerging in vivo therapies aim to directly deliver CRISPR/Cas
systems to specific cells within the body, providing a more
accessible and scalable approach to gene therapy.
Gene is disrupted Gene has new sequence
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