caa a22 4500 475787064 CHVBK 20180406123701.0 cr unu---uuuuu 170329e20001201xx s 000 0 eng 10.1007/PL00014411 doi (NATIONALLICENCE)springer-10.1007/PL00014411 Nevin Norman C. Department of Medical Genetics, Belfast City Hospital, Lisburn Road, Belfast BT9 7AB, Northern Ireland, UK e-mail: nc.nevin@bch.n-i.nhs.uk Tel: +44-28-90263873; Fax: +44-28-90236911, IE aut What has happened to gene therapy? [Elektronische Daten] [Norman C. Nevin] 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. Springer-Verlag Berlin Heidelberg, 2000 Key words Gene therapy nationallicence Gene Therapy Advisory Committee nationallicence Monogenic disorder nationallicence RNA/DNA oligonucleotides nationallicence https://doi.org/10.1007/PL00014411 text/html Onlinezugriff via DOI 1 research-article jats NATIONALLICENCE 856 40 https://doi.org/10.1007/PL00014411 text/html Onlinezugriff via DOI NATIONALLICENCE 100 1- Nevin Norman C. Department of Medical Genetics, Belfast City Hospital, Lisburn Road, Belfast BT9 7AB, Northern Ireland, UK e-mail: nc.nevin@bch.n-i.nhs.uk Tel: +44-28-90263873; Fax: +44-28-90236911, IE aut Metadata rights reserved Springer special CC-BY-NC licence nationallicence BK010053 XK010053 XK010000 NATIONALLICENCE NATIONALLICENCE NL-springer