3 ms·
Absolutely not. Scaling is the whole problem with gene therapy. If we could scale it we would have had it in 1999. In that year, a man was injected with lentiv
by mjfl 10y ago
Absolutely not. Scaling is the whole problem with gene therapy. If we could scale it we would have had it in 1999. In that year, a man was injected with lentivirally carried genetic therapy to cause his liver cells to break down ammonia (they weren't before and he couldn't eat certain foods, lived an uncomfortable but livable life). He died due to the immune response. His name was [Jesse Gelsinger](https://en.wikipedia.org/wiki/Jesse_Gelsinger https://en.wikipedia.org/wiki/Jesse_Gelsinger). It froze gene therapy funding for 10 years. Siddharta Mukerjee has a really good discussion on it in his book, The Gene.
Scaling gene therapy is THE problem of gene therapy. Not editing genes themselves.
- dekhn 10y ago(I know the answer, but) Why would the death of a single person from a clinical trial freeze an entire category of funding? Surely, many people die in clinical trials of other types of medicine (caused by the treatment) and that doesn't cause the trials to be shut down. Immunotherapy of cancer wouldn't be a viable treatment if subjected to the same level of regulation as gene therapy.