The Committee for Advanced Therapies' of the European Medicines Agency Reflection Paper on Management of Clinical Risks Deriving from Insertional Mutagenesis

被引：29

作者：

Aiuti, Alessandro ^{[1
,2
,3
,4
]}

Cossu, Giulio ^{[5
,6
]}

de Felipe, Pablo ^{[1
,7
]}

Galli, Maria Cristina ^{[1
,8
]}

Narayanan, Gopalan ^{[9
]}

Renner, Matthias ^{[1
,10
]}

Stahlbom, Axel ^{[1
,11
]}

Schneider, Christian K. ^{[5
,12
,13
]}

Voltz-Girolt, Caroline ^{[1
]}

机构：

[1] European Med Agcy, Gene Therapy Working Party Comm Adv Therapies, London E14 4HB, England

[2] San Raffaele Telethon Inst Gene Therapy, I-20132 Milan, MI, Italy

[3] Bambino Gesu Pediat Hosp, Univ Dept Pediat, Rome, Italy

[4] Tor Vergata Univ, Rome, Italy

[5] European Med Agcy, Comm Adv Therapies, London E14 4HB, England

[6] UCL, Dept Cell & Dev Biol, London WC1E 6BT, England

[7] Agencia Espanola Medicamentos & Prod Sanitarios, Madrid, Spain

[8] Ist Super Sanita, I-00161 Rome, Italy

[9] Medicines & Healthcare Prod Regulatory Agcy, London SW1W 9SZ, England

[10] Paul Ehrlich Inst, D-63225 Langen, Germany

[11] Lakemedelsverket, Med Prod Agcy, S-75103 Uppsala, Sweden

[12] Danish Hlth & Med Author, DK-2300 Copenhagen, Denmark

[13] Twincore Ctr Expt & Clin Infect Res, D-730625 Hannover, Germany

来源：

HUMAN GENE THERAPY CLINICAL DEVELOPMENT | 2013年 / 24卷 / 02期

基金：

英国医学研究理事会;

关键词：

CELL GENE-THERAPY; LENTIVIRAL VECTOR; RETROVIRAL VECTORS; MOUSE MODEL; INTEGRATION; ACTIVATION; DESIGN; TRIAL; TRANSPLANTATION; GENOTOXICITY;

D O I：

10.1089/humc.2013.119

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

In the European Union, the Committee for Advanced Therapies of the European Medicines Agency takes the lead in the scientific assessment for marketing authorization applications for advanced therapy medicinal products, which include gene therapy medicinal products, somatic cell therapy medicinal products, and tissue-engineered products. The Committee for Advanced Therapies also takes the lead in defining the scientific framework for the quality, nonclinical and clinical development of such products. This reflection paper represents the Committee's current thinking on management of clinical risks deriving from insertional mutagenesis. A multidisciplinary approach to insertional mutagenesis is provided. This reflection paper has been adopted by the committee in its April 2013 meeting.

引用

页码：47 / 54

页数：8

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