A new form of genetic editing cured deafness in a new successful trial…
The Broad Institute of MIT and Harvard. A team of researchers has used a technique called base editing to cure deafness in mice.
The Broad Institute previously was instrumental in developing CRISPR genetic editing technology. CRISPR corrects genetic mutations by using an enzyme to cut out an unwanted genetic mutation and replacing it with healthy DNA.
Base Editing is different in that it doesn’t cut the DNA. Instead, it can “rewrite” the actual letters in the DNA sequence.
DNA is the building block of life. It is the hereditary material contained in every cell of our body.
All strands of DNA consist of a sugar, a phosphate group, and a base pair. The sugar and phosphate group are identical in every strand of a person’s DNA. They are the “backbone.” The base pair determines what genetic information is expressed.
In DNA, there are only four bases that make up a pair. They are adenine (A), cytosine (C), guanine (G), and thymine (T). Adenine always bonds with thymine to form a pair. And guanine always bonds with cytosine to form a pair.
The only naturally occurring base pairs in DNA are A-T and G-C. When we talk about “letters,” this is what we are talking about. Below is a visual:
DNA Base Pairs
Source: How Stuff Works
Now, Genetic Mutations occur when one of these bases is transcribed incorrectly – that is, when the letters don’t match up properly.
Base editing technology corrects these mutations by “rewriting” the letters so that they match up correctly. In other words, base editing can change any single letter to any other.
When it comes to a certain kind of deafness in mice, the condition is caused by a single mutation. That makes it relatively easy to cure with base editing.
Using base editing, the team proved that the mice could hear sounds as quiet as 60 decibels, which is the level of a normal conversation. That’s not 100% hearing, but it is still a remarkable achievement. The mice could hear again.
This technique could easily be applied to humans. That’s the next step for the team at Broad.
Well, base editing is an exciting technology that’s on the rise. Base editing is now where CRISPR was six or seven years ago. It is not a matter of being better or worse than CRISPR. This is another tool to help cure all human disease.
We’re finding that with some genetic mutations, different enzymes work better than others. The same will likely be true about using base editing versus CRISPR depending on the situation.
We will see some exciting developments in base editing technology in the last half of this year. This is another powerful tool when it comes to eradicating all human disease.
https://www.broadinstitute.orgBroadInstitute is a mission-driven community that brings together researchers in medicine, biology, chemistry, computation, engineering, and mathematics from across MIT, Harvard, and Harvard-affiliated hospitals, along with collaborators around the world