Trials have demonstrated the feasibility of gene therapy in correcting the
gene defect in monogenic disorders such as severe combined immune deficienc
y and cystic fibrosis, but there are still obstacles and ethical issues to
overcome.
Conclusion With appropriate research, better delivery strategies and adhere
nce to good standard clinical research and practice, gene therapy research
will lead to clinical implementation in monogenic and multifactorial disord
ers including cancer, neurodegenerative disorders and vascular disease.