Amyotrophic lateral sclerosis (ALS)

Progeria
Cataract
Cystic fibrosis
Wolf man syndrome
6000
Diseases caused by genetic
mutations

95%
With no approved
therapeutic options
The Genome Editing Era
CRISPR/Cas9: the revolution

Hussein Sabit, PhD

Professor of Cancer Genetics, College of Biotechnology,
Misr University for Science & Technology
CRISPR/Cas9
CRISPR timeline
Yoshizumi Ishino

John van der Oost Virginijus Siksnys

Francisco Mojika

Feng Zhan
Philippe Horvath
Nobel Prize in Chemistry (2020)
 CRISPR/Cas-9
Clustered, Regularly Interspaced, Short,
Palindromic Repeats
Origin of CRISPR
 CRISPR ..
the gift of mother Nature!
We are inspired by Bacteria!

3900 million years

 CRISPR is a defense in
prokaryotes
90% 40%
in Archaea In Bacteria
How it works?
CRISP R locus on the Bacterial Chromosome

Spacers Spacers
CAAAACCTTGGTAAGCTTTGTCGTGTCGAGTTTTG CAAAACCTTGGTAAGCTTTGTCGTGTCGAGTTTTG
35 bp 35 bp

Palindromic Repeat Palindromic Repeat

Only one in 10 Million Bacterial cells survives
viral infection
Avoiding self-destruction

un e
A u toimm
r t of
A so se a se
Di
The outcome
DSB: Double Strand Breaks
HDR: Homology-directed
Repair
NHEJ: Non-homologous End
Joining
How it works?
Applications
of CRISPR
The new toolbox to deal with our genome!
Gene drive
Geometric series
Geometric series
Candida albicans
 Gene drive
Pushing a species to the point of
extinction or improve it?
Chemical Surgery
A step forward
C-base editing
Curing Cancer
Eradicating HIV
Huntington Disease
Triple-muscled animals
Hornless Holstein
Changing flower color
Micro Pigs
 First-ever
CRISPR meal
Designer babies
Editing Human
embryos
CRISPR Therapeutics and Vertex
Pharmaceuticals conducted the first
clinical trial with CRISPR/Cas9 to treat β-
thalassemia.
 Mice transplanted with human hematopoietic stem cells that have an HIV
receptor gene, CCR5, disrupted by CRISPR/Cas9 allows the animals to ward off
HIV infection.
Wheat without gluten for Coeliac disease
 TRACE
Temporal Recording in Arrays by
CRISPR Expansion
Original GIF Reconstructed GIF from bacteria
 Molecular Recorder

Original GIF Reconstructed GIF from bacteria

CRISPR modifies the Epigenome!
 ways to use CRISPR in vivo:
§ Gels and creams (to treat HPV)
§ Drinkable or edible CRISPR (to fight bacteria)
§ Ear injections (to treat hearing loss)
§ Skin grafts (to manage type 2 diabetes)
§ Ex vivo therapy (to treat treating sickle cell disease.)
Even RNA could be edited

CRISPR/Cas13
Is it?
 Ecological
disequilibrium
CRISPR could change life by changing
the ...
This might create chimeric human!
The Cyborg!
 The fear
of the unknown!
thank you