Photo: Bret Hartman / TED
Prof. David R. Liu, Director of the Merkin Institute of Transformative Technologies in Healthcare and Vice-Chair of the Faculty at the Broad Institute of Harvard and MIT, in his 2019 TED Talk, addressed a potential way to cure impossible-to-treat genetic diseases.
Diseases like progeria, muscular dystrophy or sickle cell anemia, have been for many years, sources of extensive studies by scientists around the globe, since the gene-editing technique CRISPR was innovated. CRISPR is a molecule, functioning as a pair of scissors, that can be used to cut DNA strands.
"Almost everything can affect changes in a human body (i.e. sunlight, cigarettes, food or alcohol). The most common change is often caused by a simple swap of letter (or base) such as C, with a different letter such as T, G or A", according to Prof. Liu. "The swaps of letters are referred to as Point Mutations. Genetic Diseases are inherited from parents or developed at a very early age; they are usually caused by certain point mutations disrupting important capabilities in cells or cause the cells to misbehave in harmful ways."
Base Editing was developed in an effort to correct point mutations that cause genetic diseases. Using CRISPRs' amazing capability to be programmed to search and cut specific sequences in the DNA, Prof. Liu and his team of students, applied this principle to create what he calls Base Editors. The particularity of Base Editors is that, instead of cutting the desired DNA strand like CRISPRs, they simply convert one base to another base without disrupting the rest of the gene. Base Editors were engineered to make it possible to convert Cs into Ts at a specified location in the genome.
After over three years of developing the Base Editing technique, Base Editors have now become widely used by scientists and researchers around the world. "Its more than 6000 Base Editor blueprints that have been distributed to more than 1000 researchers around the globe and hundreds of scientific projects, using Base Editors, have been published..." according to Prof. Liu.
Although Base Editors are still new to be used on human beings, they have been used on animals to correct a point mutation that contributes to genetic diseases and in plants to produce better crops. A future, where we are able to cure genetic diseases in human beings, is more than ever very close.