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European Biopharmaceutical Review

Cut and Paste

The successful sequencing of the human genome in 2003 was a momentous accomplishment and has turned out to be a gift that keeps on giving. A renewed understanding of biology and pathophysiology has advanced the knowledge and utility of many existing fields, such as rare diseases, genetic disorders and cancer, to name but three. As a result, there has been an explosion of new techniques to diagnose, prevent and treat pathologies. This singular event has also led to the spawning of new areas like next-generation sequencing and gene-editing. The latter is a disruptive emerging technology that has the potential to alter the way we view genetic or gene-related diseases, changing many from being lethal to chronic at worst and curable at best.

CRISPR-Cas9


Heraclitus, a Greek philosopher, is credited with saying that Ďchange is the only constant in lifeí; in science, this continues to prove true. In the 90s, gene therapy was hailed as the next big thing at the cutting edge of science. Revolutionary as it was, it has stuttered and spluttered, failing to break into the realms of mainstream therapies. Gene therapy involves using a neutered virus (known as a viral vector) to deliver a synthetic functioning copy of a gene into cells. The trouble is that there is no control over where the gene will end up in the three billion DNA base pairs, hence it is a rather blunt instrument.

While gene therapies were still experiencing teething problems, two bacteriologists serendipitously discovered a process that would progress genetic science. They observed that bacteria, when defending themselves from a virus, would replicate the virusís DNA and attach a key enzyme called Cas9. This would then unlock the DNA of the virus, break and disable it, thereby rendering the virus impotent. Mimicking this clever process from nature, clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 technology was born.


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Jimmy Muchechetere is a Healthcare and Biotech Research Analyst at Investec, having joined the financial industry in 2011 at Williams De BroŽ (now Investec Wealth & Investment) as a Research Intern. He is responsible for the equity research of all pharma as well as other healthcare and biotech companies across the globe. Jimmy also oversees the smooth running of the research departmentís international service and is a registered medical doctor with seven yearsí experience.
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