Genome Editing

 

Columbia University and Broad Institute have been researching a new tool, designed for gene alterations.  Unlike the CRISPR, which is designed to fix DNA sequences through cutting, the evoCAST is designed to insert entire genes that are healthy into precise locations.  With the evoCAST, diseases like cystic fibrosis and hemophilia could be treated more smoothly and reliably.  So, how does it work?  The evoCAST is based on CRISPR technology, so the genes are able to "jump" to new locations in the genome.  Jumping genes are also referred to as transposons.  Genetic diversity can also be a byproduct of genes moving from place to place in the genome.  In order to make this usable for medicine, researchers used the PACE technique, or the Phage-assisted continuous development, where enzymes had to go through rounds and rounds of fast-paced evolution.  This technique raised the efficiency levels in human cells to a place where it could potentially be used for real-world gene therapy.  Treatments like this could be revolutionary to those afflicted with diseases like hemophilia and cystic fibrosis, which are caused by many different mutations.  Back before the evoCAST, the CRISPR had to be used, and to use it for treatment, a custom drug had to be designed for every mutation found.  With this new technology, one therapy could be used to insert an entire healthy copy of a gene into the genome.

Picture/article: https://www.cuimc.columbia.edu/news/sternberg-evocast-gene-editor 

Second article: https://www.broadinstitute.org/news/technology-brings-new-precision-to-genome-editing