CRISPR: Can it really offer us hope?

There is a new kid on the block in the world of biology. One which has instigated a huge upheaval in biomedical laboratories across the world. CRISPR or clustered regularly interspaced palindromic repeats is a form of gene editing technology which not only brings hope to the table but a heap of controversy too.


File:CRISPR-Cas9 mode of action.png
Mode of action of CRISPR/Cas9.This file is licensed under the Creative Commons Attribution-Share Alike 4.0 International license.




What is CRISPR?

In short, CRISPR is a novel scientific technology which can correct errors within the genome as well as being able to switch specific genes on or off. The genome is the complete genetic material which makes up an organism and contains its DNA. CRISPR isn’t ground breaking in terms of allowing scientists to edit the genomes of different organisms, we have been able to do that for years but it does allow it to be completed cost effectively and efficiently. CRISPR works using two vital components: a guide RNA and CRISPR associated protein-9 (Cas9). A complex of the guide RNA and the Cas9 are delivered into the cell, allowing the genome of the cell to be cut at desired locations. Ultimately, this allows new genes to be added or existing genes to be removed, in terms of disease this is a crucial advantage CRISPR has to offer.

CRISPRs potential uses…

A promising use for CRISPR is the treatment of genetic disorders caused by a single gene mutation examples include Duchenne’s Muscular Dystrophy (DMD), Cystic Fibrosis (CF) and haemoglobinopathies such as sickle cell anaemia or thalassaemia’s. A study by Schwank and colleagues (2013) successfully used the CRISPR Cas9 system to correct the most frequent mutation which is responsible for CF in intestinal organoids. Once the mutation had been fixed, the group showed the function of the otherwise defected CF transmembrane conductor receptor (CTFR) had been repaired.

However, there are many who have reservations about gene editing technology such as CRISPR. Campaign groups have expressed their fears particularly regarding genetically modified (GM) crops. Editing crops to produce sustainable food for an ever growing population which also have greater pest resistance are being condemned by groups such as Greenpeace.

Although, there is opposition to CRISPR it may also have its advantages. Fears surrounding “designer babies” are rife in daily newspapers but in reality, would this be the best use of such a technology? Is there really a need to modify embryos for advantageous traits? Earlier in the week, research published in the journal Nature showed groups from the US and South Korea had corrected a mutation responsible for hypertrophic cardiomyopathy in preimplantation human embryos. The teams allowed the embryos to develop for 5 days before stopping the experiment. Already questions are being asked about the morality of this type of treatment.

Hypertrophic cardiomyopathy is a common condition caused by a single gene mutation and if you carry the gene then you have a 1 in 2 chance of passing the disease onto your children. Research like this has been carried out before but resulted mosaic embryos (mix of diseased and healthy cells) and mutations occurring in other cells. However, the latest research has so far not caused the technical issues experienced in the past. This type of modification is not about to become the norm and only worked in 72% of the embryos used.

What it does highlight though is the potential application of CRISPR and the advantages it could and is bringing to biology. CRISPR is undoubtedly catapulting science into a golden era of research.


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