Translating the genomics revolution: The need for an international gene therapy consortium for monogenic diseases

Molecular Therapy

Volumn 21, Issue 2, 2013, Pages 266-268

  Tremblay, Jacques P ^a   Xiao, Xiao ^b   Aartsma Rus, Annemieke ^c   Barbas, Carlos ^d   Blau, Helen M ^e   Bogdanove, Adam J ^f   Boycott, Kym ^g   Braun, Serge ^h   Breakefield, Xandra O ⁱ   Bueren, Juan A ^j   Buschmann, Michael ^k   Byrne, Barry J ^l   Calos, Michele ^e   Cathomen, Toni ^m   Chamberlain, Jeffrey ⁿ   Chuah, Marinee ^o   Cornetta, Kenneth ^p   Davies, Kay E ^q   Dickson, J George ^r   Duchateau, Philippe ^s   Flotte, Terence R ^t   Gaudet, Daniel ^u   Gersbach, Charles A ^v   Gilbert, Renald ^w   Glorioso, Joseph ^x   Herzog, Roland W ^y   High, Katherine A ^z   Huang, Wenlin ^aa   Huard, Johnny ^x   Joung, J Keith ⁱ   Liu, Depei ^ab   Liu, Dexi ^ac   Lochmüller, Hanns ^ad   Lustig, Lawrence ^ae   Martens, Jeffrey ^af   Massie, Bernard ^ag   Mavilio, Fulvio ^ah   Mendell, Jerry R ^ai   Nathwani, Amit ^aj   Ponder, Katherine ^ak   Porteus, Matthew ^e   Puymirat, Jack ^a   Samulski, Jude ^al   Takeda, Shin'Ichi ^am   Thrasher, Adrian ^an   Vandendriessche, Thierry ^o   Wei, Yuquan ^ao   Wilson, James M ^ap   Wilton, Steve D ^aq   Wolfe, John H ^ap   Gao, Guangping ^t   more..

^a Université Laval   (Canada)

^b UNIVERSITY OF NORTH CAROLINA   (United States)

^c LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^d SCRIPPS RESEARCH INSTITUTE   (United States)

^e STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^f Department of Obstetrics Gynecology   (United States)

^g CHILDREN S HOSPITAL OF EASTERN ONTARIO   (Canada)

^h AFM   (France)

ⁱ MASSACHUSETTS GENERAL HOSPITAL   (United States)

^j CIEMAT   (Spain)

^k ÉCOLE POLYTECHNIQUE DE MONTRÉAL   (Canada)

^l UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

^m UNIVERSITY OF FREIBURG   (Germany)

ⁿ UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^o VRIJE UNIVERSITEIT BRUSSEL   (Belgium)

^p INDIANA UNIVERSITY SCHOOL OF MEDICINE   (United States)

^q UNIVERSITY OF OXFORD   (United Kingdom)

^r UNIVERSITY OF LONDON   (United Kingdom)

^s Arvid Wallgrens Backe 20   (France)

^t UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

^u UNIVERSITÉ DE MONTRÉAL   (Canada)

^v Duke University   (United States)

^w Biotechnlogy Research Institute   (Canada)

^x UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

^y UNIVERSITY OF FLORIDA   (United States)

^z UNIVERSITY OF PENNSYLVANIA   (United States)

^aa SUN YAT SEN UNIVERSITY CANCER CENTER   (China)

^ab INSTITUTE OF BASIC MEDICAL SCIENCES   (China)

^ac UNIVERSITY OF GEORGIA   (United States)

^ad NEWCASTLE UNIVERSITY   (United Kingdom)

^ae UNIVERSITY OF CALIFORNIA   (United States)

^af UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

^ag NATIONAL RESEARCH COUNCIL CANADA   (Canada)

^ah GÉNÉTHON   (France)

^ai OHIO STATE UNIVERSITY   (United States)

^aj UNIVERSITY COLLEGE LONDON   (United Kingdom)

^ak WASHINGTON UNIVERSITY   (United States)

^al DUKE UNIVERSITY MEDICAL CENTER   (United States)

^am NATIONAL INSTITUTE OF MENTAL HEALTH   (Japan)

^an UNIVERSITY COLLEGE LONDON   (United Kingdom)

^ao SICHUAN UNIVERSITY   (China)

^ap UNIVERSITY OF PENNSYLVANIA   (United States)

^aq UNIVERSITY OF WESTERN AUSTRALIA   (Australia)

more..

Author keywords

[No Author keywords available]

Indexed keywords

AUTOLOGOUS STEM CELL TRANSPLANTATION; COST EFFECTIVENESS ANALYSIS; DEGENERATIVE DISEASE; GENE SEQUENCE; GENE THERAPY; HUMAN; KIDNEY DISEASE; LETTER; MONOGENIC DISORDER; NEUROLOGIC DISEASE; PLURIPOTENT STEM CELL;

BIOMEDICAL RESEARCH; GENETIC THERAPY; GENOMICS; HUMANS; INTERNATIONAL COOPERATION;

EID: 84873333610     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2013.4     Document Type: Letter

References (4)

1. Takahashi, K and Yamanaka, S (2006). Induction of pluripotent stem cells from mouse embryonic and adult fbroblast cultures by defned factors. Cell 126: 663-676.
2. Gurdon, JB, Byrne, JA and Simonsson, S (2003). Nuclear reprogramming and stem cell creation. Proc Natl Acad Sci USA 100 (suppl. 1): 11819-11822.
3. Gaudet, D, Méthot, J, Déry, S, Brisson, D, Essiembre, C, Tremblay, G et al. (2012). Effcacy and long-term safety of alipogene tiparvovec (AAV1-LPL(S447X)) gene therapy for lipoprotein lipase defciency: an open-label trial. Gene Ther, e-pub ahead of print 21 June 2012.
4. Mavilio, F (2012). Gene therapies need new development models. Nature 490: 7.