3 ms·
This is absolutely incredible! Does anyone know if this can help GSD? My cousin has it and it’s an absolutely horrible disease, her childhood was robbed and he
by seibelj 8y ago
This is absolutely incredible!
Does anyone know if this can help GSD? My cousin has it and it’s an absolutely horrible disease, her childhood was robbed and her parents' lives upended https://en.m.wikipedia.org/wiki/Glycogen_storage_disease https://en.m.wikipedia.org/wiki/Glycogen_storage_disease
- Scoundreller 8y agoMy guess is that it would be difficult because you have to kill off/weaken the existing problematic cells to allow the new ones to take over with these therapies. This is manageable when you’re treating a white blood cell disease as you can live temporarily without these cells and they regenerate frequently. But if all you need are some cells to produce more of an enzyme and can produce it in sufficient amounts to manage the existing cells producing the bad enzyme, you might be in luck.
- DrAwdeOccarim 8y agoMaybe mRNA therapy could help your cousin? There are a number of companies working on using mRNA to treat genetic diseases.