Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases

Author:

Tremblay Jacques P,Xiao Xiao,Aartsma-Rus Annemieke,Barbas Carlos,Blau Helen M,Bogdanove Adam J,Boycott Kym,Braun Serge,Breakefield Xandra O,Bueren Juan A,Buschmann Michael,Byrne Barry J,Calos Michele,Cathomen Toni,Chamberlain Jeffrey,Chuah Marinee,Cornetta Kenneth,Davies Kay E,Dickson J George,Duchateau Philippe,Flotte Terence R,Gaudet Daniel,Gersbach Charles A,Gilbert Renald,Glorioso Joseph,Herzog Roland W,High Katherine A,Huang Wenlin,Huard Johnny,Joung J Keith,Liu Depei,Liu Dexi,Lochmüller Hanns,Lustig Lawrence,Martens Jeffrey,Massie Bernard,Mavilio Fulvio,Mendell Jerry R,Nathwani Amit,Ponder Katherine,Porteus Matthew,Puymirat Jack,Samulski Jude,Takeda Shin'ichi,Thrasher Adrian,VandenDriessche Thierry,Wei Yuquan,Wilson James M,Wilton Steve D,Wolfe John H,Gao Guangping

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference4 articles.

1. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors;Takahashi;Cell,2006

2. Nuclear reprogramming and stem cell creation;Gurdon;Proc Natl Acad Sci USA,2003

3. Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPL(S447X)) gene therapy for lipoprotein lipase deficiency: an open-label trial;Gaudet;Gene Ther,2012

4. Gene therapies need new development models;Mavilio;Nature,2012

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