Gene Therapy: Applications and Ethics

This assignment delves into the promising field of gene therapy, examining its applications in treating various diseases like cancer and genetic disorders. It also critically analyzes the ethical considerations surrounding gene therapy, including genetic enhancement and the potential for unforeseen consequences. The discussion encompasses historical developments, current research advancements, and future perspectives in this rapidly evolving field.

Mutations

Introduction
The process of introduction of changes in the current gene structure or base unit of DNA
is mutation. Unlike the comic world fantasy changes, mutation has certain serious scientific
aspects which will be discussed in the presented essay. The effects and ethical considerations of
gene therapy and mutation process will be presented for giving deep understanding regarding in
the subject.
Biochemical composition, structure and replication of DNA
Deoxyribonucleic Acid abbreviated as DNA is the basic unit that helps in decoding the
informative content like inherited traits, ancestral details, genes and their structure.
Chromosomes form the nuclei of cells which are present with a thread like structure in higher
organisms like human beings (Charlesworth and Charlesworth, 2010). DNA is present in
chromosomes with surroundings of proteins. The foundations of genetics is dependent on the
functions and behaviour of DNA inside a cell. Entire process of evolution of human beings and
other organisms has been decoded with help of studies and researches conducted on DNA. All
the instructions regarding genes, development or functioning of an organism are carried by this
unit.
The biochemical composition of DNA is made up of chains of two strands of the unit that
are continuous in nature and have nucleotides or monomer units. The components of these
monomer units with either of the four bases i.e. cytosine (C), guanine (G), adenine (A) or
thymine (T) and deoxyribose or phosphate as the sugar. A covalent bond is shared between these
bases and the sugar acts like a backbone to the structure. Continuous and helical structure make
the double strands of DNA (Duarte and et. al., 2012).
The process of DNA replication is based on the production of two similar molecules of
DNA from one original unit. Inheritance is based on this process. The original molecule has two
strands which are separated and used as the arrangement for production of other two
counterparts. The result is obtained with production of two replicated molecules from one
individual unit (Guerrini and Falchi, 2011). In order to get precise characteristics and traits
which accurately match with the original molecule, error checking mechanisms are formulated.
Unlike the mutation process described in comic books the realistic results are far from fantasies.
The extrachromosonal DNA present in an organism is completely altered on a permanent basis
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and this automatically produces a deflective behaviour in the organism. It is a result of the errors
that occur in DNA replication process.
Different types of mutations that can take place
As depicted in the X-Men comics, mutations generate super power abilities in living
organisms which can be varied in terms of characteristics. But practical biological sciences do
not infer on such fantasies. Mutations are said to have contribution in both development and
destruction of the organism (Liang, Tang and Halliwell, 2010). The duplication process involves
recombination of large number of DNA molecules and there are differences created in the
genetic structure. There are various kinds of mutations experienced by making alterations or
changes in the original DNA structure. The chromosome abnormality or mutation is a kind of
mutation that occurs due to flaws or deformities in the pair of chromosomes in an organism.
These are further classified into deletion, duplication, inversion, insertion and
translocation. The irregularities that are witnessed in the chromosome pairs or many a times one
single chromosome is considered to be mutation. It can be due to formation of an extra or
missing or deformed part of the chromosomal DNA (Poduri, Evrony and Walsh, 2013). This
indicates that there is a variation in traits and genes of the organism with this defect as compared
to normalised behaviour of an individual. This type of mutation takes place right at the time of
cell division when an organism is growing. On the other hand, there is another type of mutation
that is caused by exposure to radiations or chemicals which change the biological composition
and structure of the DNA.
Originally used in agriculture for producing best quality produce, seeds where exposed to
heavy radiations and chemicals for getting desired traits in the targeted seed. There have been
various researches conducted on living organisms mostly animals to produce a species that are
engineered and biologically designed by introducing alterations in the basic structure of DNA
(Tenaillon and et. al., 2012). Also known as mutation breeding, this process was initially used for
getting different breeds of plants and produce in agriculture. Several accidents related to nuclear
radiations and chemical leaks have resulted in production of mutant birds, bugs and animals. The
consequences of such alterations can be fatal and disturbing to the ecology. The mutation process
has different effects on protein synthesis process.
Effect of mutations on protein synthesis
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Protein synthesis is a mechanism of generation of new proteins which is accompanied by
degradation or breakdown of the current proteins (Wainreb and et. al., 2011). The entire process
takes place in the cell itself. Generally, mutation can occur by chance i.e. either by medical errors
or by introducing a desirable change in the DNA. Genes are the mostly affected part in this
whole process because they carry the information or instructions inside the DNA. Protein
synthesis is essential for proper functioning of the biological mechanisms. There are cases where
mutations have taken place but no side effects were experienced on the other hand, many
mutations have caused development of tumour or cancer like conditions (Weitz and et. al., 2012).
Proteins completely control the chemical composition and structure of the genes. Having
such a crucial function in the organisms biological structure, protein synthesis is the formation of
new proteins by translation of RNA i.e. ribonucleic acid. The information that certain attributes
in the organism has to be changed is carried by gene. RNA is given the instructions regarding
where the changes in the code of information has to be made and where no changes are to be
introduced (Wirth, Parker and Ylä-Herttuala, 2013). Considering the case of nuclear mutation,
the chemical or nuclear radiations that are being introduced in the organism's cell structure then
two possibilities are identified. Either the targeted organism looses certain functions or gains an
extra characteristic.
The proteins which formulate information are either reduced or increased in terms of
functions. Abilities of an individual specie or splice is endangered with such a procedure.
Despite of being a beneficial research the mutation process can have severe impact over original
DNA. Organism can develop deflective characteristics which may or may not acquire the
designed quality for which entire processing was initiated (Wollert and Drexler, 2010). The
unsuccessful operation of mutation breeding can result in production of defective gene and
hence, protein synthesis is altered and affects other generations of the organism. Hence, it is
implied that movies and fictional comics like X-Men where mutation brings in super powers and
extra-ordinary abilities are all imaginary. Real time mutation experiments bring in a lot of critical
consequences which are harmful for the ecology.
Moral concerns of enhancement gene therapy/ ethical issues surrounding this technology
Gene therapy or genetic enhancement therapy is based on the transfer or modifications
that are made to the ordinary genes of an individual especially humans for gaining desirable
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traits and characteristics. It can be performed for gaining immunity towards various diseases or
in order to enhance the performance levels in a person. Often performed with help of drugs the
basic use of gene therapy is critically linked with mutation process. The mutants in X-Men
comics had undergone gene enhancement therapy that altered their gene structure permanently.
The results of this therapy in the comics were extraordinary but practically there are various
moral and ethical issues related to the process (DNA – structure, 2017).
The different kinds of researches and experimentations for successful mutation on
organisms especially animals is very unethical. The changes in the gene structure of a living
being causes severe effects on the functionality of the organism. It is like putting an innocent life
in danger for gathering scientific information and successful researches (Genetic Enhancement.
2017. The gene enhancement therapy is a technology which has given lots of benefit to mankind
and also aided human life for sustaining in the world. But the use of nuclear radiations if not
handled appropriately can cause severe drawbacks on the environment as well as on the life on
which experimentation is performed.
Despite of the fact that future generations will be safeguarded from the illness and
ailments that are caused by genetic disorders, there are chances that if gene therapy fails, the
reverse effect will be created over future organisms. Morale and ethical issues will take a serious
face only negligence or uncontrolled atmosphere is provided to the genes and DNA cells of the
subject (What physical or mental enhancements are possible through gene therapy ?. 2014).
Henceforth, it is essential to develop an experimentation in the gene enhancement theory in such
a way that no harm is caused to living organisms and the surrounding environment.
Conclusion
Through this essay, one can understand the basic structure of DNA and its contribution in
the development of an organism. Basic aspects of mutation and the principle types have been
discussed above. The effects of such mutation processes on the protein synthesis process and the
ethical considerations related to the subject have also been presented to gain an overview of
mutation and gene enhancement therapy.
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REFERENCES
Books and Journals
Charlesworth, B. and Charlesworth, D., 2010. Elements of evolutionary genetics. Greenwood
Village: Roberts and Company Publishers.
Duarte, S. and et. al., 2012. Suicide gene therapy in cancer: where do we stand now?. Cancer
letters. 324(2). pp.160-170.
Guerrini, R. and Falchi, M., 2011. Dravet syndrome and SCN1A gene mutation related‐
epilepsies: cognitive impairment and its determinants. Developmental Medicine & Child
Neurology. 53(s2). pp.11-15.
Liang, H. D., Tang, J. and Halliwell, M., 2010. Sonoporation, drug delivery, and gene therapy.
Proceedings of the Institution of Mechanical Engineers, Part H: Journal of Engineering in
Medicine. 224(2). pp.343-361.
Poduri, A., Evrony, G. D. and Walsh, C.A., 2013. Somatic mutation, genomic variation, and
neurological disease. Science. 341(6141). p.1237758.
Tenaillon, O. and et. al., 2012. The molecular diversity of adaptive convergence. Science.
335(6067). pp.457-461.
Wainreb, G. and et. al., 2011. Protein stability: a single recorded mutation aids in predicting the
effects of other mutations in the same amino acid site. Bioinformatics. 27(23). pp.3286-3292.
Weitz, I. C. and et. al., 2012. Eculizumab therapy results in rapid and sustained decreases in
markers of thrombin generation and inflammation in patients with PNH independent of its
effects on hemolysis and microparticle formation. Thrombosis research. 130(3). pp.361-368.
Wirth, T., Parker, N. and Ylä-Herttuala, S., 2013. History of gene therapy. Gene. 525(2). pp.162-
169.
Wollert, K. C. and Drexler, H., 2010. Cell therapy for the treatment of coronary heart disease: a
critical appraisal. Nature Reviews Cardiology. 7(4). pp.204-215.
Online
DNA – structure. 2017. [Online]. Available
Through:<http://www.chemguide.co.uk/organicprops/aminoacids/dna1.html>. [Accessed on 7th
March, 2017].
Genetic Enhancement. 2017. [Online]. Available
Through:<https://www.genome.gov/10004767/genetic-enhancement/>. [Accessed on 7th March,
2017].
What physical or mental enhancements are possible through gene therapy ?. 2014. [Online].
Available Through:<http://hplusmagazine.com/2014/05/29/what-physical-or-mental-
enhancements-are-possible-through-gene-therapy/>. [Accessed on 7th March, 2017].
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