GENI BIO

INDUSTRIEL

“ Genome
Editing ”
Framed by :
PR. ATTARCA
By :
 Souad Bousaboune N°15
 Narjiss Alaoui N°06
 Work plan:
Introduction
01 What do we mean by ‘genome editing’?

The context of genome

02 How does it work? Impact on research.

The goals of genome

03 What is genome editing used for? Other
applications: industrial…

Moral perspectives
04 Science as a moral enterprise, Intervening in the
genome, Intervening in the genome…
Introduction :

So what do we mean by
‘genome editing’?
 Timeline of genome editing
Cre-lox Bacterie
The Cre-lox editing CRISPR/Cas
technology was The CRISPR defense
successfully used for system is first
site-specific identified in
recombination in mice prokaryotes
1989 1998 2013

1992 2000
HR- mediated Zinc-finger nucleases CRISPR/Cas genome
targeting (ZFNs) editing
First study describing Discovery of zinc- First demonstration
genome editing via HR in finger proteins that that the CRISPR/Cas
mouce ES cells can target specific system can be used
DNA sequences for mammalian cell
genome editing
 Introduction :
This review considers the impact of recent advances in genome
editing, which have diffused rapidly across many fields of
biological research, and the range of ethical questions to which
they give rise. It was carried out by an interdisciplinary
Working Group that included expertise in science, law,
philosophy, ethics, sociology and industry. In coming to its
conclusions, the Working Group invited contributions from a
wide range of people, including through an open call for
evidence that ran from November 2015 until February 2016.

The review sets out our preliminary findings on the impact of

genome editing across different areas of biological research
and applications, and the range of questions to which this gives
rise.
What do we mean by Genome Editing?

The term ‘genome’ generally refers to the entire sequence of

Genome DNA of an organism. The genome includes genes: sequences of
DNA with specific functions that are involved in the production
of the proteins needed to carry out many biological roles. It also
editing ??!! includes regions of DNA that promote or inhibit gene activity,
and regions that do not appear to affect protein production or
function
Genome editing is the deliberate alteration of a selected DNA
sequence in a living cell. A strand of DNA is cut at a specific
point and naturally existing cellular repair mechanisms, then fix
the broken DNA strands. The way they are repaired can affect
gene function and new DNA sequences can be delivered when
the DNA is cut and act as templates for generating an altered
sequence. Genome editing techniques can be used to delete
sections of DNA or alter how a gene functions: for example, by
changing a variant that may give rise to disease to one that
Almost all cells of any living organism
(e.g. a human, animal, plant,
bacterium) contain DNA, a type of
molecule that is passed from one
generation to the next during
reproduction. DNA is involved in
many essential biological processes
including building cells and
controlling their number and type, the
production of energy, the regulation of
metabolism, and fighting disease
Goals of genome

What’s genome editing used for?

Other applications:
industrial…
 Applications of Gene Editing :
As discussed before, many scientists use gene editing technology for loss or gain of function analyses. These
approaches can be used to address a huge variety of questions - not least the role that a specific gene or
mutation plays in a biological pathway or the pathogenesis of a disease. And in much the same way that
scientists have done with preceeding technologies such as RNAi and transgenes is, a wide variety of read outs,
such as expression, localization, interaction and pathway activation, can be used to study the effect of the gene
edit.
Identifying synthetic lethal
Using gene knockouts to interactions (and potential
validate antibodies for novel drug targets) using
research or diagnostic use isogenic cell lines

Creation of an allele Creation of optogenetics

specific RNAi sensitive rat models with specific
cell lines classes of neurons that can
Studying modulators of be switched on with light
gene transcription with stimulation
endogenous reporters
 Other applications:
Industrial applications
Industrial applications of genome editing in
bacteria include the production of fossil fuel
alternatives, food additives and flavorings,
antibiotics, herbicides and vaccine
production. Potential benefits of this
method of production include fast turn-
around times and the use of cheap supply
materials (even waste products in some
cases).
Military applications
apk
Military interest in the applications of genome
editing may be focused on its potential for
defense purposes, for example to counteract
the release of a harmful substance to a
population.
There is also speculative interest in the
possibility that genome editing may one day
have a role to play in the selecting or
enhancing of military personnel in relation to
genetic susceptibility to disease or improved
Amateur applications
the comparatively low cost, ease of use and
availability of online kits making use of
genome editing technologies mean they are
accessible to amateur users outside of
regulated industrial and academic
environments. Scientists, school and
undergraduate students (e.g. in biology
competitions), and others with an interest in
biological research and the possibilities –
whether potentially beneficial or harmful –
raised by genome.
Biosafety
Genome-edited organisms (as with all
genetically modified organisms or GMOs) pose
a possible risk of harm to those handling them,
and to others or to natural ecosystems if they
are released or escape from controlled
environments. there are concerns about how
these can be managed outside of regulated
environments, like in countries with less well-
developed structures, and by individuals who
are not disciplined with regard to health
The context of genome

How does it work?

Impact on research…
 How does Genome Editing work ?

 Genome editing uses a type of enzyme called an engineered nuclease which

cuts the genome in a specific place.
 Engineered nucleases are made up of two parts:
• A nuclease part that cuts the DNA.
• A DNA-targeting part that is designed to guide the nuclease to a
specific sequence of DNA.
 After cutting the DNA in a specific place, the cell will naturally repair the
cut.
 We can manipulate this repair process to make changes or edits to the
DNA in that location in the genome.
Types of Genome Editing :
A nuclease enzyme is engineered to cut at a
specific location in the DNA.
Text Here
Easy to change
colors, photos and
After cutting the DNA with the engineered nuclease,
Text. the cell’s normal DNA repair (The process by which a
cell recognises and corrects damage to its DNA )
machinery will recognise the damage and join the two
cut ends of DNA back together.

This simple repair process is not 100 per cent perfect

and often a few bases are lost or added around the
site of the cut when it is repaired.
 

This small change mutation in the DNA will affect the

function of that section of DNA, which mean a gene
doesn’t function properly or doesn’t function at all
Removal of a section of DNA :

To remove a section of DNA,

01 nucleases are engineered that make
cuts in the DNA either side of the
section that we want to remove.

After the engineered nucleases cut

the DNA, the cell’s normal DNA
repair machinery will recognise the
02 damage but may mistakenly join the
wrong ends of DNA together,
removing the DNA in between the
two cuts.
Insertion of section of DNA :

A natural DNA repair system can be taked to

01 insert a section of DNA into a genome by
genome editing

Normally before a cell divides, all of its DNA is

copied so that the two resulting daughter cells
02 can receive a complete copy of the genome

After the DNA has been cut, a modified piece

03 of DNA similar in sequence to the site of the
cut is introduced
Goals of genome

What do People think about

Genome Editing ?
 What people think about genome editing ?
Patient and Public Perspectives

Patient communities

Patients, patient advocates, and families of patients with genetic disorders have diverse views
on whether germline genome editing should be used to prevent or treat genetic disorders.
Some patients suffering from conditions such as huntington disease believe strongly that it
should be used to prevent people from getting genetic diseases, especially ones that do not
currently have treatment options. Others, such as those in the deaf community, do not
consider their condition to be a disability. They worry that if human genome editing becomes
widespread, persons born with genetic conditions would be less likely to be accepted in
society. Many communities generally question the idea that eliminating genetic conditions will
improve lives, especially given that those with disabilities often report a high quality of life.

The public 
 The Pew Research Center, a nonpartisan group that polls the public on a variety of topics,  conducted
a survey to understand the public's feelings about gene editing for newborns that would give them a
reduced risk of serious diseases during their lives. The survey noted that any change made could be
passed down to future generations, which could eventually change the genetic characteristics of the
population.Though they found some enthusiasm among the public about gene editing, they also found
that about two-thirds of U.S. adults have worries about the technology.

Additional findings from Pew include:

 The public is about evenly divided on whether they would

PORTFOLIO use gene editing for their own child

 The public is about evenly divided on whether gene
editing "crosses a line" and constitutes "meddling with
PRESENTATION nature," or is like "other ways we try to better ourselves"
 There are religious divides in opinions about gene editing
DESIGNED  Those that had prior knowledge about gene editing were
more inclined to want it for their own child in the given
scenario.
Thanks for your
Attention