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CRISPR- Changing Lives, Changing the Future

CRISPR- Changing Lives, Changing the Future

CRISPR, pronounced "crisper", is a biological system for altering DNA. Better known as gene editing, this technology has the potential to change the lives of everyone and everything on the planet, writes Fergus Walsh, medical correspondent to BBC. 

CRISPR was co-discovered in 2012 by molecular biologist Professor Jennifer Doudna whose team at Berkeley, University of California was studying how bacteria defend themselves against viral infection. Doudna and her collaborator Emmanuelle Charpentier, who are considered among the world's most influential scientists, discovered the natural system that can be used by biologists to make precise changes to any DNA.

Professor Doudna said, "Since we published our work four years ago laboratories around the world have adopted this technology for applications in animals, plants, humans, fungi, other bacteria: essentially any kind of organism they are studying."

When a bacterium comes under attack it produces a piece of genetic material that matches the genetic sequence of the invading virus. This piece of material in tandem with a key protein called Cas9 can then lock on to the DNA of the virus, break it and disable it. Scientists are now able to deploy the same process to insert, delete or repair DNA. It is so sensitive that they can use it to explore the billions of chemical combinations that make up code of the DNA in a cell, and to make a single key change. Crucially, it is fast and cheap, and so is accelerating all kinds of research, from the creation of genetically-modified animal models of human disease to the search for the DNA mutations that trigger illness or confer protection.

Given that the technology is just a few years old, it is not surprising that trials on patients have yet to begin but the concept is in the planning stages. The Boston biotech firm Editas Medicine is hoping to have a gene-editing treatment ready for clinical testing in 2017 to treat Leber congenital amaurosis (LCA10), a rare retinal disease that causes blindness. 

Several other biotech firms, hoping to take the technology into clinics, are working on the theory that CRISPR might be used to boost the function of the body's T cells so that the immune system is better at recognising and killing cancer. Disorders of the blood and immune system are other potential targets. Besides the ongoing development on CRISPR, two earlier forms of gene editing have already made it into the clinic.

However, some of the key scientists in this field have concerns about the potential misuse of a technology that could be used for eugenics, to create genetic discrimination. Doudna’s response on this view is that that while it is very hard to regulate the use of CRISPR technology, it is important to find a consensus about how people should proceed.
Apart from all ongoing studies, research and developments, one cloud hanging over all this effort is a big patent fight over CRISPR. On one side is Professor Doudna's team, and on the other, a group based in Boston, Massachusetts. The patent row is unlikely to prevent academic researchers from using CRISPR, but it could have a profound impact on who reaps the financial returns of this emerging technology.

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