Snip snip – good as new!
This is Sandra Tsing Loh with the Loh Down on Science.
Changes, or mutations in genes are the causes of many diseases. But what if we could use “genetic scissors” to correct a gene that was causing trouble?
Hilary Longhurst and team from the University of Auckland used this idea for Hereditary Angioedema, or H-A-E.
H-A-E is caused by a genetic mutation that leads to an overproduction of a protein called kallikrein. This leads to high levels of another protein called bradykinin which can cause fatal swelling.
They used CRISPR/Cas9 gene editing to cut pieces of the Kallikrein gene, and measured protein levels.
Results? All ten patients showed a decrease of sixty-seven to ninety-five percent of kallikrein levels! Patients also experienced an average of a ninety-five percent decrease of swelling attacks per month.
Directly correcting mutations could be the answer to cure genetic diseases!
So this summer, let’s turn those GENES into cut-off’s! No, not your mom jeans! I’ve tried that.
Reference: Longhurst HJ, Lindsay K, Petersen RS, Fijen LM, Gurugama P, Maag D, Butler JS, Shah MY, Golden A, Xu Y, Boiselle C, Vogel JD, Abdelhady AM, Maitland ML, McKee MD, Seitzer J, Han BW, Soukamneuth S, Leonard J, Sepp-Lorenzino L, Clark ED, Lebwohl D, Cohn DM. CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema. N Engl J Med. 2024 Feb 1;390(5):432-441. doi: 10.1056/NEJMoa2309149. PMID: 38294975.