2 ms·
I've learned a lot about gene therapy. The immune system is a major bottleneck to wide spread gene therapy. If you modify the genetic makeup of cells in your bo
by jostmey 6y ago
I've learned a lot about gene therapy. The immune system is a major bottleneck to wide spread gene therapy. If you modify the genetic makeup of cells in your body, the immune system will recognize the new antigens being presented by these cells and attack. Many gene therapy target point mutations that don't require significant alterations to correct that would alarm the immune system.
- f6v 6y agoIt’s also that we don’t have organism-scale models of how change in one gene will affect other body functions. We don’t know how to build these kind of models and we don’t have even 1% of the data to do that. Organoids are just emerging, but they aren’t 1:1 representation of what happens in real human.
- amelius 6y agoDon't point mutations cause proteins to fold differently? And thus causing the proteins to become globally different rather than locally different?
- jostmey 6y ago1. Most point mutations won't result in significant changes, but if we are talking about gene therapy, the specific point mutation probably did effect protein folding or function 2. Proteins in each cell are broken down into unfolded fragments and presented on major histocompatibility complexes as antigen.