What has happened to gene therapy?

Authors
Citation
Nc. Nevin, What has happened to gene therapy?, EUR J PED, 159, 2000, pp. S240-S242
Citations number
10
Categorie Soggetti
Pediatrics,"Medical Research General Topics
Journal title
EUROPEAN JOURNAL OF PEDIATRICS
ISSN journal
03406199 → ACNP
Volume
159
Year of publication
2000
Supplement
3
Pages
S240 - S242
Database
ISI
SICI code
0340-6199(200012)159:<S240:WHHTGT>2.0.ZU;2-P
Abstract
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.