Gene Therapy – On the Ethical and Legal Issues Aspect

In this day and age, gene therapy is a new and innovative method to treat and prevent diseases by injecting a gene into a patient’s cell (Ama-assn.org, 2016). As gene therapy cures disease by generating modifications in the human body, many ethical and legal problems arise (Genetics Home Reference, 2016).

Some people argue that the regulations will be difficult to control. This is because even though with laws, there is also a possibility that gene therapy may be offered in the black market. For instance, gene therapy might use for inherently associated quality such as everlasting look, personality, or physical improvement (Ndsu.edu, 2016).

Besides, the community has been talking about ethical issues about germline therapy. The inserted gene can be passed on to the future generations by using germline therapy. Thus, it could impact the growth of a foetus in unpredicted ways. People who got affected cannot choose whether to have treatment due to the fact that they are not born yet, hence it is unfair to them (Genetics Home Reference, 2016).

Moreover, religion is another concern on gene therapy. Some think that it is immoral to alter DNA (Ndsu.edu, 2016). In addition, invasion of privacy is also one of the issues. For instance, the insurance company might make it compulsory to have a genetic check-up before they issue a statement. This could cause unfairness to families with genetic diseases (Ndsu.edu, 2016).

In a nutshell, the government shall develop the solutions before releasing gene therapy in the future. I suggest that gene therapy should be practiced by the geneticists in a more ethical and legal way while treating patients.

Written by: Ke Hong Yee

References

Ama-assn.org. (2016). Gene Therapy. [online] Available at: http://www.ama-assn.org/ama/pub/physician-resources/medical-science/genetics-molecular-medicine/current-topics/gene-therapy.page? [Accessed 20 Oct. 2016].

Genetics Home Reference. (2016). What are the ethical issues surrounding gene therapy?. [online] Available at: https://ghr.nlm.nih.gov/primer/therapy/ethics [Accessed 29 Oct. 2016].

Ndsu.edu. (2016). A New Future: Gene Therapy. [online] Available at: https://www.ndsu.edu/pubweb/~mcclean/plsc431/students98/fleck.htm [Accessed 20 Oct. 2016].

Does Genetic Engineering field emerging and/or converging to influence gene therapy?

Over the years, geneticists have learned about the effects of changes in DNA. Recently, more ‘gene editing’ technologies have been discovered and developed to target gene better. This includes CRISPR which currently beats others by becoming the most reliable, cheapest and fastest ‘gene editing’ method  (YourGenome.org, 2016). So, let’s see what is CRISPR?

CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats which refers to unusual DNA sequences that help to protect organisms such as human by targeting threats like viruses and attacking them. Furthermore, CRISPR is spaced out at clear intervals, clustered and they look like short palindromes repeating themselves overly with slight variations. Besides, CRISPR has specific enzymes known as Cas which produced specifically and numbered according to their purposes at each time. How does this Cas enable the organism to defend itself successfully? This is through the binding of Cas to the viral DNA and conducts slicing at its weak spot based on the encoded information (Lacoma, 2016).

Diagram showing how the CRISPR-Cas9 editing tool works.

Image credit: Genome Research Limited.

Moreover, CRISPR- Cas 9 is a very potential treating tools in treating medical conditions, including hepatitis B, high cholesterol and even cancer. Currently, most of the proposed applications involving editing genomes of the somatic (non-reproductive) cell and indeed they have treated some life threatening cases. However, there has been a debate about the genome editing in the germline (reproductive) cell. It is deemed illegal in most countries as the changes made will pass on to the future generations which has important ethical implications (Lacoma, 2016).

All in all, it is now clearly shown that CRISPR technology’s potential exceed beyond DNA cleavage (Reis, et al., 2014). The limitation faced is only the geneticist’s imagination on the usefulness for genome locus-specific recruitment of proteins. In my opinion, the rapid progress in developing CRISPR with the aim of eventually using it to treat human diseases routinely is possible in many more years due to there is still pending researches focusing on eliminating ‘off-target’ effects. This is to prevent the different gene is targeted from the one was intended to be edited (Lacoma, 2016).

Written by: Chiew Shi Wai

References

Lacoma, T., 2016. CRISPR 101: A crash course on the revolutionary gene editing tool that’s changing the world. [Online]
Available at: http://www.digitaltrends.com/cool-tech/what-is-crispr-a-beginners-guide/
[Accessed 28 October 2016].

Reis, A., Hornblower, B., Robb, B. & Tzertzinis, G., 2014. CRISPR/Cas9 and Targeted Genome Editing: A New Era in Molecular Biology. [Online]
Available at: https://www.neb.com/tools-and-resources/feature-articles/crispr-cas9-and-targeted-genome-editing-a-new-era-in-molecular-biology
[Accessed 28 October 2016].

YourGenome.org, 2016. What is CRISPR-Cas9?. [Online]
Available at: http://www.yourgenome.org/facts/what-is-crispr-cas9
[Accessed 28 October 2016].

Genetic Engineering

This video shows how Genetic Engineering that would change everything forever where modified humans and all living things would take place.

Link: https://www.youtube.com/watch?v=jAhjPd4uNFY

Video credits: Kurzgesagt –  In a Nutshell @ Youtube

Written by: Poo Kah Wai

What are the Risks behind and Feasibility to be discovered in Gene Therapy?

According to a notable death in gene therapy trials at 1999, Gelsinger, the subject died four days after the treatment due to serious immune reaction (Mandal, 2014). However, by taking a stand on gene therapy, we need to accept and acknowledge the risks besides the benefits mentioned in the older post.

Photo credits:  http://classic-cd.com/ 

Risks included (MayoClinic, 2016): –

a.) The virus may cause infection after being introduced into the body due to their original disease-causation ability may be recovered.

b.) The wrong cells may be targeted which lead to healthy cells damaged and other illnesses. Worse, it may cause cancer too. It’s possible that the altered viruses infect other ‘innocent’ cells as not only the targeted cells will contain mutated genes.

c.) The inflammation and organ failure can happen because of unwanted immune reaction happened due to the patient’s body immune system might mistakenly detect newly introduced viruses as invaders and thus taken the wrong mechanism steps to attack them.

d.) The tumour formation could occur after the wrong insertion of a new gene in the DNA strand.

For future betterment, safer and improved viral vectors are to be licensed nowadays by several therapeutics companies like Avexis, Dimension Therapeutics, Voyager Therapeutics and Regenxbio (Chen, 2016).

Besides of the risks, what do we understand about the feasibility of gene therapy?

   Photo Credits: www.projectsmart.co.uk

There are a lot of studies show that gene therapy is feasible to cure many diseases recently, including Wiskott-Aldrich syndrome (WAS), head & neck cancer and even tooth regeneration as well (Cavazzana, 2015) (Vattemi & Claudio, 2009) (Takahashi, et al., 2013).

Viral vectors i.e.: adenoviral and retroviral vectors remain as the top gene transfer vehicles of choice, constituting a quarter among all viral vectors used in clinical trials currently. Several feasible strategies regarding cancer gene therapy have been developed, including (Vattemi & Claudio, 2009) :-

• Replacement of tumour suppressor gene function;
• Blockage of dominant oncogene function;
• Oncolytic virus therapy, which kills tumour cells selectively but not normal cells;
• Genetic pro-drug activation therapy
• Genetic immunomodulation

These converging approaches in cancer gene therapy are often to be combined in therapies to further amplify potential therapeutic effects (Vattemi & Claudio, 2009). On the other hand, in a study of gene therapy has also proven that gene therapy enables substantial restoration of immune function which prolonged clinical benefit to severe WAS patients (Cavazzana, 2015). On top of that, In Vivo gene therapy has already been discovered as a suitable approach to tooth regeneration by stimulation of the 3rd dentition (Takahashi, et al., 2013).

On the whole, gene therapy used to be believed as an elegant, easy and highly-feasible approach that could treat diseases at its root, but it turned out way more complicated that we envisioned. Thus, the geneticists should use gene therapy safely by minimising the risk and possible side-effects.

Written by: Chiew Shi Wai

References

Cavazzana, M., 2015. Study shows feasibility of using gene therapy to treat rare immunodeficiency syndrome. [Online] Available at: https://www.eurekalert.org/pub_releases/2015-04/tjnj-ssf041615.php [Accessed 18 October 2016].

Chen, C., 2016. A Pioneer of Gene Therapy on the Future of the Industry. [Online] Available at: http://www.bloomberg.com/news/articles/2016-08-04/a-pioneer-of-gene-therapy-on-the-future-of-the-industry [Accessed 18 October 2016].

Mandal, A., 2014. Gene Therapy Issues. [Online] Available at: http://www.news-medical.net/health/Gene-Therapy-Issues.aspx [Accessed 18 October 2016].

MayoClinic, 2016. Gene Therapy Risks. [Online] Available at: http://www.mayoclinic.org/tests-procedures/gene-therapy/details/risks/cmc-20243698 [Accessed 18 October 2016].

Takahashi, K. et al., 2013. Feasibility of Gene Therapy for Tooth Regeneration by Stimulation of a Third Dentition. [Online] Available at: http://www.intechopen.com/books/gene-therapy-tools-and-potential-applications/feasibility-of-gene-therapy-for-tooth-regeneration-by-stimulation-of-a-third-dentition [Accessed 18 October 2016].

Vattemi, E. & Claudio, P. P., 2009. The feasibility of gene therapy in the treatment of head and neck cancer. [Online] Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2640478/ [Accessed 18 October 2016].