Defeat Duchenne Canada: 2023 Research Grant Update - Dr. Dongshen Duan

Thanks to our donors and supporters, Defeat Duchenne Canada has been the leading funder of Duchenne muscular dystrophy research in Canada for more than 29 years, granting $17.73 million to date.
Hear an update from Dr. Dongshen Duan at the University of Missouri in Columbia, MO, who is in his third and final year of funding from Defeat Duchenne Canada on his funded project: ‘Super AAV for DMD Gene Therapy.’
Learn more at: defeatduchenne.ca/research-we-fund

[@jaysimoes3705]

I think SGT-003 uses a capsid that is engineered to be much more effective to target human muscles and in fact far less the liver (unwanted). So there are already capsid being developped out of current vectors and this will only get better, which is very usefull since for one we could reduce the side effects (less and less of target action) and lower the dose needed (smaller dose, better targeted = higher efficacy adn lower sides).
Good to see more researchers going into that direction. Also I wonder how research is developping in bypassing the human defense mechanism so we could re-use some vectors and trigger an immune-response! Because with most current microdystrophine (but also micro utrophine and GALGT2) it is one shot and then it can't be re-used because immunesystem responses. Since muscles develop etc current microdystrophine therapies are probably not functional through life but gradually wears off. Either we need to get rid of capsids or we need to modulate the immunesystem. I wonder if research is done on that possibility?