Today we want to tell you about a very special girl called Alyssa. Because she is the first person in the world whose life has been saved by Base Editing, As we think that this will change all our lives, for the better, very soon, we’re going to run a slightly longer blog today. Because we think that you, as citizens of the educated world would like to know a little more about what it is. And what it represents in the bigger scheme of things.
The Story Alyssa had a terminal form of leukaemia which wasn’t responding to conventional therapies. Not even at London’s Great Ormond Street, one of the world centres for paediatric medicine. So doctors staked all on Base Editing a technique that’s so very new it hasn’t really hit the trial stages yet. Alyssa’s story is well covered here by Sarah Knapton of the Daily Telegraph. Plus Sarah gives a pretty good explanation of the technique
CRISPR-Cas- 9, The Daddy of Base Editing Well, Base Editing is really a specialised form of CRISPR-Cas-9, as astute readers of this blog will remember (LSS 25 7 20,16 7 22 et al) CRISPR was a natural defence mechanism of bacteria to help them snip the DNA of hostile viruses. Clever scientists purloined this and combined it the Cas-9 protein system to let them snip the DNA wherever they chose. The problem was that it could break open the double chain of the DNA molecule.
And so to Base Editing The base editing refinement avoids this problem. It is now possible to introduce single nucleotide changes at any point on the genome you want. without breaking the DNA molecule of the patient. The potential to overcome many diseases is now there. Here’s a couple of worthy, if slightly heavy, guides to the whole thing
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7721651/
https://horizondiscovery.com/en/gene-editing/base-editing/advantages#:~:text=Base%20editing%200a%2
The Future? The question you ask is a fair one-“how long before this all becomes routine?” It’s early days yet, even for CRISPR. And base editing has barely reached clinical trials. This article by Bridgit Balch for AAMC news gives som eideas of the sorts of applications we might see
https://www.aamc.org/news-insights/future-crispr-now
The implications The world is now divided into two hostile blocks. The uneducated and the educated. The former despise learning, science and reason. Perhaps their most eloquent spokesperson was British politician Michael Gove, who famously declared that society had no further need of experts. They live in the comfort of their beliefs, but have no idea how to cure diseases, or make any other type of useful advance.
Experts, like those who developed CRISPR and Base editing belong to the educated block. The people who know that all learning is provisional. Which is why there needs to be much more of it. Then there could be more things like base editing
#crispr #base editing #michael gove #leukaemia
Learning, Science and Society 