Scientists at MIT and Harvard have discovered two gene editing techniques to fix mutations that cause diseases like cystic fibrosis and Duchenne muscular dystrophy.
Both diseases, and about half all human genetic disorders, are caused by mutations in single letters in the human genome, in which an ‘A’ appears where there should be a ‘B.’
The newly-developed gene editing systems can target the smallest units of our DNA or RNA to undo the mutation that causes cystic fibrosis.
One system edits DNA in the genome itself, while the other targets RNA, which transports genetic messages for making proteins.
The editing systems work in living cells, and if researchers can find ways to deliver them to human patients safely and effectively, they could be used to reverse the mutations that cause more than 15,000 genetic diseases.
Both DNA and RNA contain four base components: adenine, thymine, guanine and cytosine.
Cystic fibrosis is caused by an inherited genetic mutation that leads to abnormal mucus production in the lungs and digestive system. The thicker-than-normal mucus builds up in and blocks airways.
The disease is rare, affecting about 200,000 people in the US each year. It can be managed with breathing machines, inhalers and medications, but some affected by it will eventually need lung transplants. There is no cure for cystic fibrosis and it can be fatal.
Cystic fibrosis could be prevented or corrected if only there were a ‘G’ in the genome where the disease’s victims have an ‘A.’
With the new gene editing technologies from the Broad Institute of MIT and Harvard, scientists could rewrite the part of the genome or its messenger that spells cystic fibrosis.
The research group that created a DNA base editor, led by Dr David Liu, are calling their breakthrough a ‘molecular machine.’ Their gene editing system is technically called the Adenine Base Editor, or ABE.