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The recommendations for health maintenance, disease prevention, diagnosis, and treatment that we receive throughout our lives are largely based on our health characteristics. In addition, healthcare providers have always strived to make their practices more effective. For example, by experimenting with different treatments, observing, and sharing results and improving the efforts of previous generations. In the past, the possibilities were limited, but now technologies such as precision medicine are emerging.

Catching up with Moore

Advances in computing power and genetics and the growing availability of (health) data offer an opportunity to make accurate personalised patient care a clinical reality (Hodson, 2016). The first human genome was sequenced in 2001. After more than a decade and at a cost of about US$3 billion, Genome sequencing has become much faster and cheaper (Wetterstrand, 2020). This cost reduction led to accelerated innovation in genomics, enabling research on a larger scale.

Many genomes can now be sequenced in a single day for about $1,000 each. Many claims have been made, including by the National Human Genome Research Institute (NHGRI), that genomics outperforms developments in computing as measured by Moore’s Law (see Figure below).

This law states that the speed of computers, measured by the number of transistors that can be placed on a single chip, will double every year or two and is considered the engine of the electronic revolution (Mollick, 2006). This means that computers are increasingly providing computing power to analyse the vast amounts of data generated by DNA sequence analysis or electronic health records (EHR).

Development and possibilities

This development has led genome sequencing to enter medical practice, particularly to diagnose rare conditions where conventional techniques have failed. For example, cancer, neurodegenerative diseases and rare genetic disorders take a huge toll on individuals, families and societies as a whole. Many of these conditions can be prevented through lifestyle changes, such as improving weight and diet, or reducing alcohol consumption. However, many are also caused by ethnic, racial, or familial factors (Bresnick, 2018). Discovering how a person’s genetics affect his or her chance of developing or surviving a particular condition would fundamentally revolutionise the way health care providers approach the practice of medicine.

As former President of the United States Barack Obama said in 2015: “I want the country that eliminated polio and mapped the human genome to lead a new era of medicine, one that delivers the right treatment at the right time”. In that same year President Obama launched the Precision Medicine Initiative (PMI). Meanwhile, the global healthcare industry is slowly shifting towards a proactive and predictive approach, using algorithms for predicting risk with a higher degree of precision in certain cancers and heart diseases from available clinical data (Ehteshami Bejnordi, 2017). Although current approaches to precision medicine in oncology have been fruitful, they require better integration and utilisation of available resources to inform sustainable and effective drug development and clinical care (Marabella, 2021). These resources consist of large quantities of genetic, clinical, social, lifestyle and preference data across broad, heterogenous populations. In turn algorithms can mine knowledge from the accumulated data. To this vast store of personal data, add clinical data captured by EHRs and DNA data captured through whole-genome sequencing, and precision care becomes realisable (Baker, 2019).

More customised healthcare instead of a one-size-fits-all approach, which is the case these days, could reduce the burden of disease by personalisation, and thus the cost of preventable health care for all. In the case of the latter, the healthcare industry must make it accessible to everyone. It is also considered that certain populations will not benefit from this innovative technique because some treatments will not work for those populations, or because they will be unaffordable. Like a tailored suit, it is not for everyone but the ones who fit will benefit.

References

Hodson, R., 2016. Precision medicine. Nature Portfolio. Available at: https://www.nature.com/articles/537S49a
[Accessed 29 September 2021].

Wetterstrand, K.A., 2020. The Cost of Sequencing a Human Genome. National Human Genome Research Institute (NIH). Available at: https://www.genome.gov/about-genomics/fact-sheets/Sequencing-Human-Genome-cost
[Accessed 29 September 2021].

Mollick, E.R., 2006. Establishing Moore’s Law. Institute of Electrical and Electronics Engineers: Annals of the History of Computing. Available at: https://www.researchgate.net/journal/IEEE-Annals-of-the-History-of-Computing-1934-1547
[Accessed 30 September 2021].

Bresnick, J., 2018. What Are Precision Medicine and Personalized Medicine?. HealthITAnalytics. Available at: https://healthitanalytics.com/features/what-are-precision-medicine-and-personalized-medicine
[Accessed 30 September 2021].

Ehteshami Bejnordi, B. et al., 2017. Diagnostic Assessment of Deep Learning Algorithms for Detection of Lymph Node Metastases in Women With Breast Cancer. National Library of Medicine: National Center for Biotechnology Information. Available at: https://pubmed.ncbi.nlm.nih.gov/29234806/
[Accessed 1 October 2021].

Marabella, C., 2021. Novel Immunotherapy Options Prompt Paradigm Shift in NSCLC Treatment. OncLive. Available at: https://www.onclive.com/view/novel-immunotherapy-options-prompt-paradigm-shift-in-nsclc-treatment
[Accessed 1 October 2021].

Baker, D.B., 2019. Precision Medicine: The Promise and the Challenges. The Healthcare Information and Management Systems Society (HIMSS). Available at: https://www.himss.org/resources/precision-medicine-promise-and-challenges
[Accessed 1 October 2021].

Figure ‘Cost per Human Genome’ credits: https://www.genome.gov/about-genomics/fact-sheets/Sequencing-Human-Genome-cost
Featured image credits: Vincent Yau

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Since the discovery of DNA in the mid-19^th century and the complete sequencing of the human genome in 2003, genome (or gene) editing has made great strides in the biological field. A decade later in 2015, scientists and later Nobel Prize winners Emmanuelle Charpentier and Jennifer Doudna pioneered the revolutionary gene editing technique CRISPR. CRISPR gene editing uses an enzyme that works like molecular scissors to alter/edit targeted sections of DNA and turning genes on or off without altering their sequence (Balch, 2021).

“The ability to cut DNA where you want has revolutionized the life sciences,” said Pernilla Wittung Stafshede, member of the Nobel chemistry committee. Clinical trials are under way to use the technique to treat sickle cell anaemia, hereditary blindness, and cancer. The two scientists along with others in the field, have launched a generation of biotechnology companies focused on developing techniques to achieve these goals (Ledford & Callaway, 2020).

The emergence of a new method

With dozens of clinical trials currently in progress, CRISPR is just getting started. Meanwhile one company is making its way to rewrite the future of genetics disease. Tessera Therapeutics, a Massachusetts based biotechnology start-up has spent the past few years developing a new class of ‘molecular manipulators’ capable of doing lots of things CRISPR can do and some that it can’t. They call this method ‘gene writing’. Despite CRISPR’s therapeutic potential, the technology does have its limitations. It is useful for deleting problematic genes, but it is less effective at replacing them. This means that only certain inherited conditions can be treated this way (Jimenez, 2021). It is also better suited for editing genes in the lab, outside the body, than in living organisms (Houser, 2020). The possibility of editing (or rather writing) genes resulted in a team of researchers led by Harvard to successfully treat sickle cell disease in mice. This advancement could one day lead to a possible cure of the deadly inherited blood disorder that affects more than 300,000 newborns each year (Siliezar, 2021).

“DNA being the code of life as we know it, the opportunity to be able to make modifications with very high precision to a subset of cells in your body is going to be applicable to diseases in every therapeutic area,” said Tessera CEO Geoffrey von Maltzahn. The gene writing approach is based on mobile genetic elements, or MGEs, a class of genes that turns out to be the most abundant category of genes in nature (Al Idrus, 2021). Recent work reveals that many organisms use MGEs for specialised functions, one that depends on its ability to move around the genome and modifying the DNA sequence in the process (Jhonsa, 2020).

To accelerate its development, Tessera attracted over $230 million in financing in 2020 (Novak, 2021). This surely contributes to Tessera’s mission of delivering on the full potential of genetic medicine. This investment indicated genuine interest in the potential of gene writing and will accelerate the company’s ability to position multiple therapeutic programs for clinical development. Using computational and high-throughput laboratory platform has enabled the team to design, build, and test thousands of engineered and synthetic MGEs for writing and rewriting the human genome.

RNA-based gene writing could go where CRISPR-based editing can not and one day Tessera hopes their method will surmount the limitations of gene editing and gene therapy and cure genetic diseases by rewriting DNA.

References

Balch, B., 2021. A conversation with Jennifer Doudna, PhD, developer of CRISPR gene-editing technology. Association of American Medical Colleges (AAMC). Available at: https://www.aamc.org/news-insights/conversation-jennifer-doudna-phd-developer-crispr-gene-editing-technology
[Accessed 26 September 2021].

Ledford, H. & Callaway, E., 2020. Pioneers of revolutionary CRISPR gene editing win chemistry Nobel. Nature Portfolio. Available at: https://www.nature.com/articles/d41586-020-02765-9
[Accessed 26 September 2021].

Jimenez, D., 2021. Gene writing: The future of genetic medicine?. Pharmaceutical-technology.com. Available at: https://www.pharmaceutical-technology.com/features/gene-writing-future-genetic-medicine/
[Accessed 27 September 2021].

Houser, K., 2020. Gene writing: A new type of genetic engineering. Freethink. Available at: https://www.freethink.com/science/gene-writing
[Accessed 27 September 2021].

Siliezar, J., 2021. New gene-editing technique shows promise against sickle cell disease. The Harvard Gazette. Available at: https://news.harvard.edu/gazette/story/2021/06/gene-editing-shows-promise-as-sickle-cell-therapy/
[Accessed 27 September 2021].

Al Idrus, A., 2021. Tessera Therapeutics scores $230M to ramp up ‘gene writing’ tech to cure disease. Fierce Biotech. Available at: https://www.fiercebiotech.com/biotech/tessera-therapeutics-scores-230m-to-ramp-up-gene-writing-tech-to-cure-disease
[Accessed 27 September 2021].

Jhonsa, R., 2020. Tessera Therapeutics Aims to Rewrite DNA with New Age Gene Manipulators. Gene Online. Available at: https://www.geneonline.com/tessera-therapeutics-aims-to-rewrite-dna-with-new-age-gene-manipulators/
[Accessed 27 September 2021].

Novak, J., 2021. Tessera Therapeutics Attracts Over $230M in Series B Financing to Advance ‘Gene Writing’ – A New Category in Genetic. Bloomberg. Available at: https://www.bloomberg.com/press-releases/2021-01-12/tessera-therapeutics-attracts-over-230m-in-series-b-financing-to-advance-gene-writing-a-new-category-in-genetic
[Accessed 27 September 2021].

Featured image credits: Vincent Yau