N C Nevin1. 1. Department of Medical Genetics, Belfast City Hospital, Northern Ireland, UK. nc.nevin@bch.n-i.nhs.uk
Abstract
UNLABELLED: Trials have demonstrated the feasibility of gene therapy in correcting the gene defect in monogenic disorders such as severe combined immune deficiency and cystic fibrosis, but there are still obstacles and ethical issues to overcome. CONCLUSION: With appropriate research, better delivery strategies and adherence to good standard clinical research and practice, gene therapy research will lead to clinical implementation in monogenic and multifactorial disorders including cancer, neurodegenerative disorders and vascular disease.
UNLABELLED: Trials have demonstrated the feasibility of gene therapy in correcting the gene defect in monogenic disorders such as severe combined immune deficiency and cystic fibrosis, but there are still obstacles and ethical issues to overcome. CONCLUSION: With appropriate research, better delivery strategies and adherence to good standard clinical research and practice, gene therapy research will lead to clinical implementation in monogenic and multifactorial disorders including cancer, neurodegenerative disorders and vascular disease.