THE OPPORTUNITIES AND CHALLENGES OF GENOME EDITING – Essay Sample

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Introduction

The advent of precision genome editing has enabled scientists to modify living organisms with great precision and speed. The ability to edit genes has been in existence for decades and modifying genes is a common laboratory practice. The increased concern and attention in the field is due to the ability of present day technology to allow for precise genome editing at a high scale and speed. TALENs, Zinc-finger nucleases and now CRISPs-Cas9 system have been a revolution to scientific discovery. Genome editing can be applied to crop plants, laboratory organisms, disease vectors and domestic animals but it comes with different consequences and complexities. Application of this technology to the human germline must be closely monitored to avoid both social and scientific considerations and consequences of introducing heritable genes to the human population. In this paper I will evaluate the possibilities, challenges and ethical considerations of genome editing. I believe it is ethically justifiable to use genome editing techniques so as to improve the quality of life in all aspects of nature sciences.

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Opportunities

Genome editing tools such as CRISPR-Cas9 help in targeting specific disease genes responsible for causing diseases in certain genetic disorders. The deleterious and disease causing genes are targeted and their structure changed which causes changes in the germline with the intention of passing on to the coming generation for complete elimination of the genes. For somatic cells, gene editing which is undertaken at different clinical stages is an idea with great potential in the field of therapeutic development. Chinese researchers used CRISPR-Cas9 to eliminate human β-globulin gene from a human embryo. Mutations of β-globulin gene are responsible for causing β-thalassaemia, a dreadful blood disorder (Shalem, Sanjana & Zhang, 2015). However, the research was not done to completion because of the ethical concerns that were raised due to the research. Junjiu Huang who was leading the research gave assurance to the science community that that the embryo they used were ‘non-viable’ and had been collected from various fertility clinics. The embryos had been fertilized by two sperms and formed triponuclear zygotes which could not grow and result into a live birth. The ongoing genome editing technologies at different stages of clinical development are constrained to modifying genetic materials obtained from somatic cells. Due to the unpredictable outcomes raised by genome editing, a number of scientists are of the opinion that cure obtained from genetic editing techniques should be restrained to genome editing of the somatic cells. Genome editing results to heritable changes in the genetic makeup of embryos, and therefore could have undesired effects to the coming generations (Shalem, Sanjana & Zhang, 2015). In addition, unethical use of the genetic engineering techniques could emanate from editing of genes obtained from viable human embryos.

CRISP-Cas9 originated from type II CRISP-Cas systems which makes it possible for the bacteria to develop an adaptive immunity to invading plasmids and viruses. When they penetrate the cell of bacteria, plasmids and viruses enable the CRISPR system to integrate short viral DNA molecules into the CRISPR locus (Lanphier, Urnov, Haecker, Werner & Smolenski, 2015).

With his novel version of CRISPR-Cas9, Feng Zhang in 2013 opened the possibility of using genome editing for therapeutic purposes. This technique makes it possible for researchers to increase identification of disease causing genes and enhances therapy development to eliminate the mutated gene (Cox, Platt & Zhang, 2015). Because of its unrivaled genetic specificity, researchers are using this genome editing technology to enhance discoveries of cancer biology (Ali, 2014). Various models of cancer have been developed using the technique. However, the CRISPR-Cas9 model reflects better for disease in humans (Li et al., 2013).

Challenges

Some researchers in the science community have called for a for a pause in the use of human genome editing while others have termed it to be unethical not to use this technology which has the capability to eradicate genetic diseases and improve lives of plants and animals species in the fields of agriculture and biology (Cox, Platt & Zhang, 2015).

During their research they detected off-target mutations in the gene. These unintended genome mutations occur when CRISPR-Cas9 cleaves DNA sequences analogous to the intended sequences of DNA. These genetic mutations can turn out to be a disaster because they can lead to cell transformation and death. The Chinese researchers discovered that the frequency of the mutations were much higher than those seen in human adult cells or mice. Because of some limitations, Edward Lanphier argued that the research had to be halted until the broad discussions on the direction of the research had been concluded. On the bright side, off-target genetic mutations can be minimized by incorporating the latest CRISPR-Cas9 engineered by Yang L, et al. It has a high efficiency in targeting of site-specific genes modified by hiPSC clones.

The associated costs of using genome editing technology are only affordable by families from rich countries. Developing countries cannot afford it and this gives advantage to children born and raised in developed countries.

By use of CRISPR-Cas9, human embryo genome editing could lead to undesired effects on the coming generations. It could also be helpful for non-therapeutic modifications. The procedure will present the potential of losing eugenics and human diversity. Recently Yoshimi et al., were successful at changing color of the coat of a rat which suggest the potential of achieving a change in pigmentation in human beings vie embryo genome editing. Therefore, the enhancement of genes to obtain a specific appearance could lead to significant effect on mental and physical health of children because their appearance is no longer imposed by blood relationship.

Human embryo genome editing could also be a hindrance to the current research that entails editing of genes from somatic cells that has high possibilities for therapeutic development. Edward Lanphier et al., rightly points out that the concerns on the ethical considerations from genome editing of human embryos could incapacitate the promise of therapeutic development associated with making changes to somatic cells genes. Furthermore, there needs to be an open consultation concerning the best action to be taken in case of a compelling case for therapeutic benefit of genome editing (Ali, 2014).

Some scientists are in support of using CRISPR-Cas9 technology and argue that there is no need for a moratorium. The scientists argue that because the Chinese researchers avoided any ethical considerations that could have risen from their research by choosing to use non-viable embryos that could have not resulted in birth, then the research should be applauded because it is no worse than the discarding of non-viable embryos in In-vitro fertilization. Therefore, the calls for a moratorium have no justification. If people find using abnormal embryos that could otherwise have been discarded at the blastocyst stage then it will never be possible for scientists to find out if using it would be better or worse to use a normal embryo? The associated genetic disease is more painful than the side effects because individuals who have genetic diseases will reproduce and their offspring will be at a high risk of inheriting an undesired gene that leads to a genetic disorder (Witzany, 2010).

Conclusion

Genome editing presents great possibilities to development of new medicine, personalized medicine and human genetic modifications. It has however raised caution flags and it remains a cautionary tale. The glamour of technological and scientific advancement can make us oblivious of the ethical implications of these advancements. Some researchers have concerns that genome editing has crossed the redline and has many challenges. The research by Huang and his team on CRISPR-Cas9 was not a complete success because of the off-target mutations. The technology could consequently be used in non-therapeutic research such as creation of ‘designer babies’ by removing qualities undesired by the society and using desired ones in their place. The genome technology should however not be a hindrance to the therapeutic development involved in genome editing of somatic cells which is an area with great promises.

References

Ali, M. (2014). Understanding Cancer Mutations by Genome Editing. Uppsala: Acta Universitatis Upsaliensis.

Cox, D., Platt, R., & Zhang, F. (2015). Therapeutic genome editing: prospects and challenges. Nature Medicine, 21(2), 121-131. http://dx.doi.org/10.1038/nm.3793

Lanphier, E., Urnov, F., Haecker, S., Werner, M., & Smolenski, J. (2015). Don’t edit the human germ line. Nature, 519(7544), 410-411. http://dx.doi.org/10.1038/519410a

Li, D., Qiu, Z., Shao, Y., Chen, Y., Guan, Y., & Liu, M. et al. (2013). Heritable gene targeting in the mouse and rat using a CRISPR-Cas system. Nature Biotechnology, 31(8), 681-683. http://dx.doi.org/10.1038/nbt.2661

Shalem, O., Sanjana, N., & Zhang, F. (2015). High-throughput functional genomics using CRISPR–Cas9. Nature Reviews Genetics, 16(5), 299-311. http://dx.doi.org/10.1038/nrg3899

Witzany, G. (2010). Biocommunication and natural genome editing. Dordrecht: Springer.

Yang, L., Grishin, D., Wang, G., Aach, J., Zhang, C., & Chari, R. et al. (2014). Targeted and genome-wide sequencing reveal single nucleotide variations impacting specificity of Cas9 in human stem cells. Nature Communications, 5, 5507. http://dx.doi.org/10.1038/ncomms6507

Yoshimi, K., Kunihiro, Y., Kaneko, T., Nagahora, H., Voigt, B., & Mashimo, T. (2016). ssODN-mediated knock-in with CRISPR-Cas for large genomic regions in zygotes. Nature Communications, 7, 10431. http://dx.doi.org/10.1038/ncomms10431

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