ABE

Morphing undoes Mutation

Undesirable mutations often cause disease. Responsible for these mutations are the Dinky Amigos who make up your DNA. They gallivant about within the double helix and sometimes end up in the wrong place, giving the wrong instruction to your body and causing health problems.

Scientists have been manipulating the position of the Dinky Amigos for years. Rather more recently the currently leading technology, CRISPR, was developed to do the same thing. Gene editing using CRISPR can be more specific, but, not perfect.

Enter the Base Editor. It combines all the positives of the CRISPR gene editing system with less side effects (mostly extra mutations). Using some of the lesser known characteristics of the Dinky Amigos, the Editor can be used to replace one Dinky Amigo with another.  The undesirable Dinkie on one strand of DNA is altered to something else. This morphed Dinkie then pairs up with the desirable Dinkie on the opposing DNA strand. This replaces a mutation. Gene editing using this system can be more precise and focused.

As you might expect, Dinky Amigos don’t always relinquish their place in a line-up that easily (if they did there would be even more mutation problems!). It has to be done with cunning and stealth. Simple replacement will not work as efficiently. One Dinky Amigo must morph into another one. Crispin is much more susceptible to this cunning. He can be morphed relatively easily into Tristan.  Tristan much prefers to pair up with Alina and will ignore any advances Gina might make.  So a Crispin-Gina mutation-causing pair can be altered to a Tristan-Alina un-diseased coupling relatively easily.

Inosine_Morph

Alina is not so easy to hoodwink, however. Replacing her with Gina is not as straightforward. Luckily,  David R. Liu of Harvard University is not a man to take no for an answer. He has come up with a persuasive technique known as the Adenine Base Editor (ABE). First he takes Alina’s pony tails and ties them up. Then replaces her blue side bow with a red hair clip. Her middle clip is replaced with a bow.  He gives her another hair clip. This time a blue one for the other side. He re-names her Ina (short for Inosine).  Swap a bow for an earring, alter a necklace and before she has realised it is just a bit of harmless fun, Alina has morphed into Gina! Gina obviously then eschews Tristan in favour of Crispin and the edit has been made.

There are thousands of Alina-based single point mutations causing disease in humans.  Base Editing has already been used in a variety of organisms including human embryos. Much more practice is needed, but this editor could maybe herald the next big breakthrough for disease therapy.