A Simple Task for CRISPR

Single gene diseases


Mon, Jun 19th, 2017 11:00 by capnasty NEWS

The Guardian has a book review on A Crack in Creation, written by the biochemists who led the breakthrough on the gene editing revolution. The article quickly reviews CRISPR's involvement with yogurt, potential for easily curing single-gene diseases (cystic fibrosis, sickle-cell anaemia and muscular dystrophy) and its incredible easy-to-use power. Words of caution are not missed.

For now the most exciting potential medical application is in single gene diseases, such as cystic fibrosis, sickle-cell anaemia and muscular dystrophy. This is the simplest possible task for Crispr. Just one base has to be corrected out of the 3bn and it’s not a needle in a haystack: Crispr can find and cut and repair it. Sickle-cell anaemia is caused by a faulty haemoglobin gene, so blood can easily be withdrawn from the body, the gene edited and returned to the body. But this approach demands extreme caution. Genes often have multiple effects and the sickle-cell gene is known to protect against malaria. So if you fixed the sickle-cell gene in the African population (where it is prevalent) there would be many new cases of malaria. But then Crispr can probably fix that, too; other researchers, with Gates Foundation funding, are urgently tackling that problem. There is hardly an area of medicine that could not benefit from Crispr, and on the fringe there is the Jurassic Park fantasy, kept tenuously alive by the work of Crispr’s other great name, George Church at Harvard, who is editing the elephant genome to create a creature more like a woolly mammoth.



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