CRISPR and Conscience: Shaping
Immunity, Shaping Ethics
Exploring the consequences of gene editing for immunotherapy
Dharm Gajjar 11B1
T
he science behind gene editing
beholds a plethora of possibilities and
solutions towards achieving
immunotherapy. The emergence of such
treatments has the capability to revolutionise
the field of immunology by modifying immune
cells for therapeutic purposes. However there
are many complexities that arise when utilising
such technology and I aim to discuss this
throughout this article.
What is Genome editing?
Genome editing is the procedure that involves
changing the specific sequencing of genomes
within an organism which allow genetic
material to be added, removed, or altered at
particular locations in the genome. This can
be used to achieve a change specific traits or
physical characteristics such as eye colour and
disease risk that are expressed in an
individual. Altering DNA within an organism
can be used to acquire desired characteristics
or traits, serving a range of purposes and
significance in treating disease, which
scientists are attempting to harness by gene
therapy.
Development of Gene therapies: CRISPR-Cas9
Gene therapies are under extensive
development, involving the use of gene
editing to treat disease in humans that have
underlying conditions deriving from genetic
mutations such as cystic brosis or
polydactyly. A recent technology that has
been adopted is CRISPR-Cas9, which is short
for clustered regularly interspaced short
palindromic repeats and CRISPR-associated
protein 9. This technology has been adapted
from a defence system that bacteria tend to
use which naturally edits genomes. Upon the
infection of a virus, bacteria isolate small
fi
sections of the virus’ DNA and insert it into its
own DNA using a particular sequence to
create subsections of DNA known as CRISPR
arrays. When infected with the virus again,
these CRISPR arrays start producing RNA
corresponding to the virus’ DNA, signalling
Cas9 enzymes which cut up the DNA of the
virus, essentially disabling it.
This method of gene therapy has been
implemented in humans. Cas9 enzymes are
able to target specific DNA within a sequence,
binding and cutting it. The process is carried
out by producing RNA specific to the DNA
that needs to be altered. The RNA is then
introduced to the DNA, where Cas9 enzymes
are directed to the section of DNA, where it
can be cut out or be engineered to insert a
new DNA sequence. This can determine and
modify gene expression in humans which can
be used to modify mutations and to switch off
DNA if required. Once part of the DNA
sequence has been cut off, scientists can alter
the structure of the sequence by adding a
different segment of DNA to it.
Conventionally, the Cas9 is used more often
however the Cpf1 enzyme can also be used
too, serving the same function as the Cas9
enzyme. Such developments in science and
technology can be used to treat diseases such
as haemophilia, cystic fibrosis, sickle cell
disease, polydactyly, etc, and do so a lot
more efficiently and cheaply compared to
other gene editing methods such as TALEN or
ZFN technologies.
Concerns/Implications when using genome
editing technologies
Altering human genomes have many ethical
concerns associated alongside it. The practice
of gene editing usually occurs in somatic
 cells, which are cells other that egg and sperm
cells. However, gene editing that occurs in
sperm and egg cells, known as germline cells,
may be perceived as unethical and a method
of altering human life forms. This sparks the
immediate question of whether scientists
could enhance naturally occurring human
traits such as height or eye colour so that
humans can be engineered to possess desired
characteristics. CRISPR-Cas9 enables precise
modifications to the genome, but off-target
effects and unintended consequences remain
a significant concern. Ensuring safety
throughout the procedure is a must as a small
mistake may have a devastating impact on the
individual.

Despite the ethical and safety concerns, gene

editing technology may have a vast potential
Figure 1 shows the CRISPR system in action
in the near future, providing immunity to
genetic complications and diseases in
organisms. Obviously the technology must be
in the right hands, as gene editing can be
used wrongly to achieve desired
characteristics within organisms, which may
have negative implications within society.
Anatomical understanding has developed
increasingly over the past few decades and as
of now, we are able to thoroughly understand
different complications within genomes and fix
them using extensive knowledge of the
genome with gene editing technologies at our
disposal.

Key words
Genome - The complete set of genes or genetic Figure 2 shows the Cas9 enzyme being
material present in a cell or organism. experimented with typically on zebrafish to
DNA - (Deoxyribonucleic acid) Contains the investigate the effects of gene editing
genetic code that is unique to every individual.
Gene Therapy - Technique that modifies a
person's genes to treat or cure disease.
Mutation - Change in the DNA sequence of an
organism.
Cystic brosis - An inherited condition that causes
sticky mucus to build up in the lungs and digestive
system.
Polydactyly - A condition in which a baby is born
with one or more extra fingers.
fi
RNA - (Ribonucleic acid) Structurally similar to DNA Synoptic Links
and is involved in the transfer of DNA and protein
GCSE
synthesis.
1. 4.6.1.4 - DNA and the genome (the
Enzymes - Proteins that help speed up the rate of
importance of understanding the full set of
reaction in an organism.
genetic material in an organism)
Gene expression - The process by which specific
genes are activated to produce a required protein.
2. 4.6.1.6 - Genetic Inheritance (how
Haemophilia - An inherited bleeding disorder in
characteristics and traits are controlled by
which the blood does not clot properly.
genes)
Sickle cell disease - A group of inherited health
conditions that affect the red blood cells.
3. 4.6.1.7 - Inherited disorders (the inheritance
Somatic cells - Any cell of a living organism other
of certain genes/alleles may cause certain
than its reproductive cells.
disorders such as cystic fibrosis or polydactyly
Germline cells - An organism’s reproductive cells.

4. 4.6.2.4 - Genetic engineering (the process

References that involves modifying the genome in an
1. Broad Institute - https://www.broadinstitute.org/ organism for desired characteristics)
what-broad/areas-focus/project-spotlight/
questions-and-answers-about-crispr# 5. 4.6.1.5 - DNA structure (how the process of
transcription and protein synthesis works)
2. Ormond KE(1), Mortlock DP(2), Scholes DT(3),
Bombard Y(4), Brody LC(5), Faucett WA(6),
Garrison NA(7), Hercher L(8), Isasi R(9),
Middleton A(10), Musunuru K(11), Shriner D(12),
Virani A(13), Young CE(3). Human Germline
Genome Editing - https://medlineplus.gov/
genetics/understanding/genomicresearch/
genomeediting/

3. National Human Genome Research Institute -

https://www.genome.gov/about-genomics/
policy-issues/what-is-Genome-Editing

4. National Human Genome Research Institute -

https://www.genome.gov/about-genomics/
policy-issues/Genome-Editing/How-genome-
editing-works

5. NHS Website - https://www.nhs.uk/conditions/

sickle-cell-disease/

6. National Human Genome Research Institute -

https://www.genome.gov/human-genome-
project