CRISPR and Conscience:

Shaping Immunity, Shaping

Ethics
Exploring the consequences of gene editing for immunotherapy
Dharm Gajjar 11B1
he science behind gene editing
sections of the virus’ DNA and insert it into its
beholds a plethora of possibilities and
own DNA using a particular sequence to
solutions towards achieving
create subsections of DNA known as CRISPR
immunotherapy. The emergence of such
arrays. When infected with the virus again,
treatments has the capability to revolutionise
these CRISPR arrays start producing RNA
the field of immunology by modifying immune
corresponding to the virus’ DNA, signalling
cells for therapeutic purposes. However there
Cas9 enzymes which cut up the DNA of the
are many complexities that arise when utilising
virus, essentially disabling it.
such technology and I aim to discuss this
throughout this article. This method of gene therapy has been
implemented in humans. Cas9 enzymes are
What is Genome editing?
able to target specific DNA within a sequence,
Genome editing is the procedure that involves
binding and cutting it. The process is carried
changing the specific sequencing of genomes
out by producing RNA specific to the DNA
within an organism which allow genetic
that needs to be altered. The RNA is then
material to be added, removed, or altered at
introduced to the DNA, where Cas9 enzymes
particular locations in the genome. This can
are directed to the section of DNA, where it
be used to achieve a change specific traits or
can be cut out or be engineered to insert a
physical characteristics such as eye colour
new DNA sequence. This can determine and
and disease risk that are expressed in an
modify gene expression in humans which can
individual. Altering DNA within an organism
be used to modify mutations and to switch off
can be used to acquire desired characteristics
DNA if required. Once part of the DNA
or traits, serving a range of purposes and
sequence has been cut off, scientists can alter
significance in treating disease, which
the structure of the sequence by adding a
scientists are attempting to harness by gene
different segment of DNA to it.
therapy. Conventionally, the Cas9 is used more often
however the Cpf1 enzyme can also be used
Development of Gene therapies: CRISPR-
too, serving the same function as the Cas9
Cas9 Gene therapies are under extensive
enzyme. Such developments in science and
development, involving the use of gene
technology can be used to treat diseases
editing to treat disease in humans that have
such as haemophilia, cystic fibrosis, sickle
underlying conditions deriving from genetic
cell disease, polydactyly, etc, and do so a lot
mutations such as cystic fibrosis or
more efficiently and cheaply compared to
polydactyly. A recent technology that has
other gene editing methods such as TALEN or
been adopted is CRISPR-Cas9, which is short
ZFN technologies.
for clustered regularly interspaced short
palindromic repeats and CRISPR-associated Concerns/Implications when using genome
protein 9. This technology has been adapted editing technologies
from a defence system that bacteria tend to Altering human genomes have many ethical
use which naturally edits genomes. Upon the concerns associated alongside it. The practice
infection of a virus, bacteria isolate small 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
in the near future, providing immunity to Figure 1 shows the CRISPR system in action
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 fibrosis - 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.
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
Germline cells - An organism’s reproductive cells.
polydactyly

References 4. 4.6.2.4 - Genetic engineering (the process

1. Broad Institute - https://www.broadinstitute.org/ that involves modifying the genome in an
what-broad/areas-focus/project-spotlight/ organism for desired characteristics)
questions-and-answers-about-crispr#
5. 4.6.1.5 - DNA structure (how the process of
2. Ormond KE(1), Mortlock DP(2), Scholes DT(3), transcription and protein synthesis works)
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