2 ms·
We don’t have a good enough delivery mechanism to get it to 99.99%+ of cells. So how well a gene therapy is really depends on how many of the cells need to be t
by yazaddaruvala 5y ago
We don’t have a good enough delivery mechanism to get it to 99.99%+ of cells. So how well a gene therapy is really depends on how many of the cells need to be targeted.
For example, increasing muscle mass is beneficial even if it hits only 25% of cells. It can also be injected directly into each large muscle group. Or specifically targeted.
Meanwhile, trying to target pancreatic cancer is really hard given it’s location, and depends on if the “bad proteins” would be transported by blood to the pancreas anyways and/or if even 0.01% of cells creating the protein is enough to sustain the problem.