This blog is about an exciting new development in disease treatment - gene editing. Our medical writers explain the basics of CRISPR, a new gene editing tool. Gene editing itself isn’t new, but CRISPR has made it easier and cheaper. For a quick summary of this blog, jump to the infographic.
CRISPR: The possibilities of gene editing
In my previous life I spent many days (and nights) toiling away furiously in the laboratory in an attempt to complete my PhD. My research area was a combination of disciplines and techniques touching on genetics and gene expression. However, it wasn’t until I moved into the area of Intellectual Property Law, that I became aware of CRISPR - a powerful gene editing technology with the potential to revolutionise the treatment of diseases as we know it. Now, as a medical writer it is massively exciting to see the theory become reality with the start of projects using the technology arriving on our desks.
CRISPR (pronounced crisper), is a powerful DNA or gene editing tool whose origin lies in the natural adaptive immunity of bacteria. It enables DNA to be cut at precise locations, allowing for its accurate and targeted renewal or replacement.
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Gene editing revolution – what is it?
The path of scientific discovery often follows a pattern of years of slow progress to a sudden breakthrough that leads to a giant leap forward in the discipline as a whole and CRISPR is the Neil Armstrong of the gene editing world (if you will). Not only will it be of massive importance in the treatment of genetic disorders and drug development, it is likely to revolutionise just about any industry in which biology and genetic technology plays a part. And it’s not only the biomedical sphere that is involved, we are already seeing the application in agriculture, targeting issues such as disease resistance, flavour, texture and nutrient profile improvement in crops.
The revolution is in the accessibility of CRISPR. There are well established genome editing tools available such as zinc-fingers nucleases (ZFN) and transcription activator-like effector nucleases (TALEN), however these have often described as cumbersome, expensive and difficult to develop. CRISPR on the other hand is much easier and cheaper to use, and within a year or two of its arrival researchers were using the techniques in labs all around the world at a fraction of the cost. Importantly, the tool is precise, accurate and targeted so compared to previous methods, CRISPR is like trading in rusty scissors for a computer-controlled laser cutter.
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A precise gene editing tool
CRISPR facilitates the more precise insertion/deletion of genes or the ability to regulate their expression, which will speed up the ability to target specific genetic variants of a condition or a disease. For example, we are already seeing it being applied in a number of promising fields of research, such as T-cell therapy targeting cancer cell antigens specific to an individual patient’s cancer.
For cancer therapies
In the U.S., the University of Pennsylvania is using CRISPR to introduce modifications to T-cells taken from the patient’s body, which are then reintroduced in modified form to attack tumour cells. Indeed, potential cancer therapies appear high on agenda with clinical trials in lung cancer, cervical cancer, oesophageal cancer, bladder cancer, and prostate cancer in the pipeline. Other diseases being targeted include HIV, sickle-cell disease and β-thalassemia to name but a few.
Looking to the future of gene editing
There are many pharma giants collaborating with CRISPR start-up companies and investing heavily in research. They obviously see the potential and cannot be left behind. With such enthusiasm, money and healthy competitiveness the future of CRISPR could see the eradication of cancer, HIV, cystic fibrosis and many other incurable diseases that not only cause much human suffering but also place a heavy burden on our healthcare systems.
A note of caution
Yes, CRISPR could be the answer to many of our healthcare challenges, however, genetic modification has always been a controversial issue – think GM foods. There are regulatory issues to consider as well as ethical issues. The regulation is lagging behind the development speed of the technology and future regulation will have huge implications for the therapeutic applications of CRISPR (as well as its use in agriculture).
Ethically there are real concerns. Jennifer Doudna (one of the breakthrough scientists of CRISPR) has expressed real concerns that science could run ahead of ethics, and despite asking scientists to refrain from germline editing (i.e. embryos, sperm, egg cells) one group has already done so - to prove that diseases may be stopped before they even develop. Highlighting that even within the science world, opinions on why and how this tool is used vary massively.
There is no doubt that CRISPR is a fantastic tool with unimaginable potential. As medical writers, we will be lucky enough to see the developments and its impact on disease treatment and drug development as it happens.
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