Gene therapy clinical trials worldwide to 2012 – an update

Journal of Gene Medicine - Tập 15 Số 2 - Trang 65-77 - 2013
Samantha L. Ginn1,2, Ian E. Alexander3,1, Michael Edelstein4, Mohammad Abedi5, Jo Wixon6
1Gene Therapy Research Unit Children's Medical Research Institute and The Children's Hospital at Westmead NSW Australia
2Sydney Medical School, University of Sydney, NSW, Australia
3Discipline of Paediatrics and Child Health University of Sydney NSW Australia
4Imperial Healthcare NHS Trust, London, UK
5Department of Laboratory Medicine, Örebro University Hospital, Örebro, Sweden
6Health Sciences Journals – Editorial John Wiley & Sons Ltd Oxford UK

Tóm tắt

AbstractTo date, over 1800 gene therapy clinical trials have been completed, are ongoing or have been approved worldwide. Our database brings together global information on gene therapy clinical trials from official agency sources, published literature, conference presentations and posters kindly provided to us by individual investigators or trial sponsors.This review presents our analysis of clinical trials that, to the best of our knowledge, have been or are being performed worldwide. As of our June 2012 update, we have entries on 1843 trials undertaken in 31 countries. We have analysed the geographical distribution of trials, the disease indications (or other reasons) for trials, the proportions to which different vector types are used, and which genes have been transferred. Details of the analyses presented, and our searchable database are available on The Journal of Gene Medicine Gene Therapy Clinical Trials Worldwide website at: http://www.wiley.co.uk/genmed/clinical. We also provide an overview of the progress being made in clinical trials of gene therapy approaches around the world and discuss the prospects for the future. Copyright © 2013 John Wiley & Sons, Ltd.

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Tài liệu tham khảo

10.1126/science.286.5448.2244

10.1016/j.ymgme.2003.08.016

10.1172/JCI35700

10.1172/JCI35798

10.1126/science.288.5466.669

10.1056/NEJMoa012616

10.5694/j.1326-5377.2005.tb06785.x

10.1016/S0140-6736(04)17590-9

10.1056/NEJMoa1000164

10.1056/NEJM200301163480314

10.1126/science.1088547

10.1038/mt.2010.228

10.1126/science.270.5235.475

10.1038/nm0502-423

10.1126/science.1070104

10.1097/ACI.0b013e32833fea85

10.1182/blood-2009-06-189209

10.1056/NEJMoa0805817

10.2353/jmoldx.2007.060081

10.1016/j.iac.2010.01.002

10.1182/blood-2006-06-031476

10.1097/MOH.0b013e328302c807

10.1016/j.clim.2009.12.003

10.1126/science.1171242

Cartier N, 2012, Haematopoietic stem cell gene therapy for X‐linked adrenoleukodystrophy, Hum Gene Ther, 23, A17

Montini E, 2012, Integration site analysis in a clinical trial of lentiviral vector based haematopoietic stem cell gene therapy for meatchromatic leukodystrophy, Hum Gene Ther, 23, A13

10.1126/science.1129003

10.1200/JCO.2010.32.2537

10.1126/scitranslmed.3002842

10.1056/NEJMoa1103849

10.1126/scitranslmed.3003425

10.1073/pnas.0807027105

10.1089/hum.2008.107

10.1056/NEJMoa0802268

10.1056/NEJMc0903652

10.1038/mt.2009.277

10.1016/S0140-6736(09)61836-5

10.1126/scitranslmed.3002865

10.1038/nm1393

10.1002/jgm.991

10.1038/nm.2088

10.1182/blood-2009-05-221606

10.1182/blood-2009-05-222760

10.1038/mt.2011.166

10.1038/mt.2010.232

10.1038/mt.2010.226

10.1038/nature09328

10.1056/NEJMoa1003548

KleinC.Effective gene therapy for children with Wiskott–Aldrich syndrome. 2010. http://wwwidw‐onlinede/pages/de/news396307%3E2010 [4 February 2013].

10.1038/mt.2010.305

NIH Recombinant DNA Advisory Committee (RAC). nd.http://oba.od.nih.gov/rdna_rac/rac_about.html[4 February 2013].

GTAC summary table of UK gene therapy research. 2010.http://webarchive.nationalarchives.gov.uk/+/www.dh.gov.uk/ab/GTAC/Publications/index.htm[4 February 2013].

Belgian Biosafety server gene therapy clinical trials information.2005.http://www.biosafety.be/GT/regulatory/GTtrials.html[4 February 2013].

Internet Portal of the German Clinical trials register (DRKS).2011.https://drks‐neu.uniklinik‐freiburg.de/drks_web/[4 February 2013].

Swiss Expert Committee for Biosafety (EFBS/CFSB) gene therapy clinical trials information.2010.http://www.efbs.admin.ch/en/topics/gene‐therapy/index.html[4 February 2013].

Dutch Ministry of Housing Spatial Planning and the Environment (VROM) GGO office. nd.http://bggo.rivm.nl/Paginas/vdb.htm[4 February 2013].

AFSSAPS public clinical trials database.2009.https://icrepec.afssaps.fr/Public/index.php[4 February 2013].

European Community clinical trials database (EudraCT).2013.http://eudract.emea.europa.eu/[4 February 2013].

European Commission Genetically Modified organisms (EudraCT). 2013.http://gmoinfo.jrc.ec.europa.eu/gmo_browse.aspx[4 February 2013].

Australian New Zealand Clinical Trials Registry (EudraCT).2013.http://www.anzctr.org.au/default.aspx[4 February 2013].

West China Medical School's Chinese Clinical Trials Registry.2011.http://www.chictr.org/en/[4 February 2013].

Japan National Institute of Public Health Clinical Trial Search. 2002.http://rctportal.niph.go.jp/en/index[4 February 2013].

World Health Organization International Clinical Trials Registry Platform.2010.http://apps.who.int/trialsearch/[4 February 2013].

10.1002/jgm.1100

10.1038/nm1358

10.1182/blood-2008-07-167510

Sack BK, 2009, Evading the immune response upon in vivo gene therapy with viral vectors, Curr Opin Mol Ther, 11, 493

Nathwani A, 2010, Early clinical trial results following administration of a low dose of a novel self complementary adeno‐associated viral vector encoding human factor IX in two subjects with severe haemophilia B. American Society of haematology Annual Meeting, Blood, 116, 114, 10.1182/blood.V116.21.248.248

Basner‐Tschakarjan E, 2011, Dose‐dependent activation of capsid‐specific T cells after AAV serotype 8 vector administration in a clinical study of haemophilia B, Mol Ther, 19, S230, 10.1016/S1525-0016(16)37175-1

10.1056/NEJMoa1108046

10.1038/nm1398

10.1182/blood-2007-03-078493

10.1182/blood-2009-04-214569

10.1016/j.cell.2006.07.024

10.1038/nprot.2007.418

10.1126/science.1151526

10.1016/j.stem.2010.08.012

10.1002/stem.201

10.2183/pjab.85.348

10.1016/j.stem.2009.05.005

10.1126/science.1172482

10.1073/pnas.0801677105

10.1126/science.1152092

10.1038/nature08129

10.1038/mt.2009.274

10.1016/j.nmd.2010.07.276

10.1016/S1474-4422(09)70211-X

10.1016/S0140-6736(11)60756-3

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Journal of Gene Medicine

Tập 15 Số 2

65-77

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