- Lack of knowledge about the disease at the DNA level
- Multiple gene effects cannot be treated today
- Affected tissue must be accessible to gene therapy
- In the case of dominant disorders, homologous recombination has to occur for successful replacement of defective gene.
- Retroviral contamination and spread in the body.
- High technical expertise required
- Many ethical questions to be answered properly
In short, gene therapy is a boon if we could treat genetic diseases in a better way as we don’t have an alternative treatment method. But the pertinent question is Do we have enough knowledge to carry out this complex treatment process?