Snipping Genes & Nobel Prize Queens
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Two scientists, France’s Emmanuelle Charpentier and American Jennifer A. Doudna, won the Nobel Prize in Chemistry today for discovering and developing a powerful tool, CRISPR-Cas9, for genome editing that has enabled relatively quick and easy modification of the building blocks of life and promises new drugs for a range of diseases.
On a fundamental level, CRISPR-Cas9 can be broken into 3 parts (CRISPR, gRNA, and Cas9). The actual molecule that does the “genome editing” is a combination of guide RNa or gRNA and Cas9, the name of an enzyme that acts like a pair of scissors that can cut DNA in two locations so that DNA can be added or removed. The gRNA guides the Cas9 to the right place in the DNA sequence to ensure that the right sequence is cut. CRISPR stands for clustered regularly interspaced short palindromic repeats and is just a category of DNA sequences that can be found in prokaryotes.
As a quick background, CRISPR-CAS is a system that already exists in bacteria. When a foreign DNA sequence is found, the bacteria’s CRISPR-CAS complex cuts up the DNA so that it cannot transcribe and take over the bacteria. This was the foundation of the idea for a CRISPR-CAS complex in humans (resulting in CRISPR-Cas9).
In 2012, Charpentier and Doudna reported that the crRNA and the tracrRNA could be fused together to create a single, synthetic guide, further simplifying the system of CRISPR-Cas9.
This Nobel Prize was special for a variety of reasons. For one, only 5 other women had ever received a Nobel Prize and there had never been 2 women to receive the prize at the same time, so today’s decision marks definite improvement in the representation of women in the highest echelons of science. For another, the majority of the time a Nobel Prize is handed out, it is usually decades after the development or finding was introduced (this is because it takes time for the impact to be quantified/qualified). In this case, it has been only 8 years.
As much as this is a new technology, it is developing rapidly. Companies like Editas Medicine Inc., Sherlock Biosciences Inc, Crispr Therapeutics, Mammoth Biosciences, and many other companies have been experimenting with CRISPR-Cas9 technology. The future is ENDLESS. By editing the genome sequence, previously unavoidable genetic diseases like Alzheimer’s or Parkinson’s could be a thing of the past. No longer would people have to dread an unavoidable future just because of their chance birth. It could lengthen the lives of millions.
Of course, there are many ethical issues. Already, a Chinese researcher, Dr. Zhang, experimented on babies, which even the Chinese government declared was illegal (when CRISPR-Cas9 was being developed, it was agreed that nobody would run human trials until it had been confirmed as being 100% safe). This was because clipping the wrong gene could result in a quick death, or even worse, a potential mutation that can be passed down through generations, become ubiquitous, and ruin mankind’s future. However, the future of biotechnology/AI seems promising with AI. Though using CRISPR-Cas9 to build superhumans or ideal babies may be far off, a future where genetic diseases are literally cut away from humanity one by one may be on our fingertips.
