A growing team of groundbreaking scientists around the world are now sharing their lab notebooks online
The idea for split-intein based SCN1A reconstitution started when I first learned of the split-Cas9 system. Long story short, packaging Cas9 with a guideRNA sequence into an AAV vector is pushing the packaging capacity of AAV vector. To circumvent this issue, the authors design a split-Cas9 system where the coding sequence for Cas9 is split Read More …
In my last posts, I have constructed a pair of split-SCN1A constructs (split- SCN1A-GFP(1-10) and SCN1A-GFP(11)). Here, I tested the functionality of these constructs by looking at whether these split- constructs reconstitute into a full-length SCN1A and generate a fluorescent signal when co-transfected in HEK293 cells, and if so, do the reconstituted SCN1A (with an Read More …
Previously, I provided a brief overview on SCN1A related Dravet Syndrome (DS) and the need for larger gene therapy vectors or alternative methods to deliver functional copies of SCN1A to affected cells in DS. The goal of my project is to explore one of these alternative methods, specifically a split-intein mediated protein trans-splicing method, that Read More …
Hello Open Sciences World! In my very first post for the SGC’s Extreme Open Sciences project, I will start by providing an overview of my project – Gene therapy for Dravet Syndrome. Dravet Syndrome is a catastrophic, rare form of infantile epilepsy. The disease onset occurs in the first year of life where children with Read More …
