The challenges
For gene therapy to be effective, a gene must reach the right place in the body and become part of the normal workings of the cells involved.
It is important:
- to find the right gene
- to target the right cells in the body
- to deliver the DNA of the required gene into these cells
- to make sure the DNA is used correctly by these cells
- that the procedure is performed safely causing no injury or harmful side-effects.
To date, gene therapy has only been performed experimentally in human clinical trials, as there are several possible adverse consequences.
To see real benefit, future techniques will need to guarantee the safety of the technique and deliver the correct gene to the correct cells in the correct tissues.