


Talk to young people today (sometimes we have to) and the first thing out of their mouths is “Cool it, Daddy-oh! CRISPR Cas-9 is so, like, square! These days, all the Real Cats are heppin’ to Base Pair Editing!”
Not so fast, young friends! For news reaches us the the Food And Drug Administration of the United States may soon give the go ahead to the first treatment using CRISPR Cas-9. We follow the story from Iker Seisdedos of El País,[1] and it’s a good one. For what the creators are offering us is nothing less than a potential, once and for all cure for sickle anaemia, that painful and debilitating disease which has afflicted so many of us for thousands of years. The companies, Vertex and CRISPR Therapeutics put their case yesterday and it looks as if the FDA will give the go ahead for the next stage of trials. Possibly as early as December.
The technique is both simple and ingenious. As every school child knows, sickle cell is caused by a defect in gene BCL11A which controls the production of vital haemoglobin proteins. The trick is to extract stem cells from the patient’s bone marrow and edit the gene using standard CRISPR Cas-9 techniques, so they can now produce the correct form. The edited cells are returned to the patient where they will take over in the marrow, and start producing the good protein. And get this-once set up, it ought to stay established for life.
How well we achieve when we use the tools of observation and reason! Compare that with the destruction and waste effected in certain parts of the world by ancient religions and emotionally driven behaviours. And ask yourself “where will my true interested best be served?”
Our link today is in Spanish. so you’ll need a translator. But if you really are a lazy monoglot old anglophone, well, here’s one just for you. [2]
#crispr cas 9 #gene editing #sickle cell anaemia #