What is a problem in administering gene therapy?

1 Answer
Oct 20, 2017

The main issues with Gene therapy is the lack of knowledge about long term effects of the therapy and the field is fraught with ethical issues.


The potential risks include:

1) An unwanted immune system response may occur which in severe cases causes organ failure.
2) It is possible that the vector may infect not just the targeted cells, but also additional cells.
3) Viral vectors carry the risk of toxicity, inflammatory response and gene control and targeting issues.
4) If the new genes get inserted into the wrong spot in the DNA, chances of tumor formation may arise.
5) Some commonly occurring disorders are affected by variations in multiple genes, which often complicate Gene therapy.

A gene cannot be inserted directly into the cells. It has to be delivered using a carrier, called as a vector.