Limitations of gene therapy?

Question

What are the limitations of gene therapy? What are the challenges of gene therapy?

Answer 1

1. Knowledge of the exact cause of disease at molecular (DNA) level.
2. Region bearing disorder must be well defined.
3. Only single gene defects can be corrected today.
4. Normal counterpart of defective gene is required for gene therapy.
5. Addition of functional gene and its random integration with genome is possible part today.
6. Potentially curable diseases are single gene defects with recessive nature.
7. Affected tissue must be accessible for gene therapy.
8. Lasting presence of gene manipulating cells after re-transplantation is important. Self- renewable cells are preferred targets so diseases involving such cells could be cured.
9. Corrected cells must have some selective advantage in vivo over affected cells.
10. Spread of retrovirus throughout body and unwanted.
11. The induction of oncogenesis as a result of provirus integration is another risk.