Life sciences A to Z - G is for gene editing (CRISPR)

Life sciences A to Z - G is for gene editing (CRISPR)

CRISPR stands for “clustered regularly interspaced short palindromic repeats”, which is a mechanism that exists naturally in bacteria, helping them to resist viruses.

It came to the world’s attention in 2012 when Jennifer Doudna and her team at the University of California, Berkeley (UC Berkeley) discovered that it could be used as the basis for a revolutionary new tool for editing genes, raising the prospect of eventually eliminating hereditary diseases once and for all. In October 2020 Jennifer Doudna and Emmanuelle Charpentier (Max Planck Institute) received the Nobel prize for chemistry for their work in developing the CRISPR/Cas9 genetic scissors. The Nobel Committee said: “This technology has had a revolutionary impact on the life sciences, is contributing to new cancer therapies and may make the dream of curing inherited diseases come true.”

CRISPR, which has been described as being like the “find and replace” function in Word, is best known for its ability to target and cut specific DNA within a cell, changing the sequence of genes. However, it can also be used to turn genes on or off without changing the sequence. Developments to the original idea, known as “base” and “prime” editing, have also allowed more precise control of the changes made, making the technology more predictable and safe.

Quicker and easier

CRISPR has transformed the area of gene editing because, compared to before, it is precise, quick, and easy to use, making it relatively inexpensive. It is widely used in research, with CRISPR-based research tools being a significant area, and a number of clinical trials have also enjoyed success in using CRISPR, particularly in relation to safety. CRISPR clearly offers great promise in medicine, although it is still early days with regard to showing that the technique will be safe and effective in the clinic.[1]

Applications of CRISPR

The applications of CRISPR currently being worked on are wide-ranging. In medicine, these include cancer therapy, virus detection, curing inherited blindness and restoring lost neurons after a stroke. CRISPR is also expected to play an important role in developing personalised medicines. In agriculture, CRISPR has been used to engineer silkworms to resist a lethal virus, to produce trans-fat-free oils and create lower-gluten wheat.

Ethical issues

Although there are many beneficial applications of CRISPR, there are inevitably also ethical issues. Particular risks revolve around human germline editing which can result in heritable changes, potentially changing the course of evolution. In China the biophysicist who claimed to have created CRISPR-edited babies was sentenced to three years in jail. The use of CRISPR in agriculture is generally less controversial, although public perceptions of genetically modified (GM) food are varied, and can include strong opposition. CRISPR may be regarded as less controversial by some because it does not involve transgenic changes, just changes to the organism’s own genes, so it arguably falls outside traditional definitions of GM food.

Patent disputes

As more real-life applications of CRISPR technology come to fruition and prove themselves, the commercial value of the technology is likely to be huge. There is significant interest, therefore, in the ownership position. Unfortunately, this is currently rather opaque. Ownership of fundamental aspects of the technology continues to be heavily disputed between two US bodies: UC Berkeley and The Broad Institute (Broad); this is despite a significant victory for Broad in the US in February 2022. Both UC Berkeley and Broad also face validity challenges from third parties in respect of the relevant patents, and it appears that UC Berkeley may have the upper hand in Europe at the moment[2]. It is to be hoped that the future licensing and exploitation of the technology is not hampered by uncertainties as to who owns what.

[1] Nature 06.01.20 – Quest to use CRISPR against disease gains ground, by Heidi Ledford

[2] Nature 17.01.20 Major CRISPR patent decision won’t end tangled dispute, by Heidi Ledford


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