3 ms·
You don't necessarily need to hit every cell in an organism to produce useful therapies. Most cells aren't going to be expressing the gene that's causing a dise
by zardo 11y ago
You don't necessarily need to hit every cell in an organism to produce useful therapies. Most cells aren't going to be expressing the gene that's causing a disease, only cells in the affected tissue or organ need to be treated. In some cases, you may not even need to get all of the affected cells, just repairing a subset could improve outcomes. (1/8 of a functioning pancreas is a lot better than 0/8)