Researcher, heal thyself: meet the scientists studying their own diseases
The post Researcher, heal thyself: meet the scientists studying their own diseases appeared first on MIT School of Science.
A couple of decades after the discovery of systems that could selectively target DNA, we're starting to see the first therapies based on gene editing. One challenge these developments have faced is safety. While we can make them pretty specific to the gene we want edited, the human genome is very large, and even rare DNA sequences can appear a couple of times by chance.
As a result, all the original gene-editing systems had known rates of what are called off-target effects, in which they simply edit the wrong sequence. This may be a low-probability event, but edit enough cellsβand therapies generally have to edit manyβand errors become inevitable.
A lot of effort has gone into finding ways to minimize or eliminate off-target edits. In a recent issue of Nature, researchers described modifying the AI protein-folding software AlphaFold to help identify key areas of gene-editing proteins responsible for off-target effects. Those areas were then modified to reduce the problems.


Β© Anton Vierietin
A couple of decades after the discovery of systems that could selectively target DNA, we're starting to see the first therapies based on gene editing. One challenge these developments have faced is safety. While we can make them pretty specific to the gene we want edited, the human genome is very large, and even rare DNA sequences can appear a couple of times by chance.
As a result, all the original gene-editing systems had known rates of what are called off-target effects, in which they simply edit the wrong sequence. This may be a low-probability event, but edit enough cellsβand therapies generally have to edit manyβand errors become inevitable.
A lot of effort has gone into finding ways to minimize or eliminate off-target edits. In a recent issue of Nature, researchers described modifying the AI protein-folding software AlphaFold to help identify key areas of gene-editing proteins responsible for off-target effects. Those areas were then modified to reduce the problems.


Β© Anton Vierietin
Researchers from the University of Cambridge have created a new DNA sequencing method called Chem-map, which can detect where and how small molecule drugs interact with the targeted genome. The method enables researchers to conduct in situ mapping of small molecule-genome interactions with unprecedented precision. Chem-map was used in the study to determine the direct [β¦]
The post DNA sequencing method lifts βveilβ from genome black box appeared first on Science Bulletin.