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Journal Article
Blood, ISSN 0006-4971, 2009, Volume 114, Issue 10, pp. 2077 - 2086
In a clinical trial for adeno-associated virus serotype 1 (AAV-1)-mediated gene transfer to muscle for lipoprotein lipase (LPL) deficiency, 1 subject from the... 
RESPONSES | AAV VECTOR | LIPOPROTEIN-LIPASE DEFICIENCY | ADENOASSOCIATED VIRAL VECTOR | LONG-TERM CORRECTION | VIRUS | BENEFICIAL MUTATION | CANINE HEMOPHILIA-B | FACTOR-IX | HEMATOLOGY | LEBERS CONGENITAL AMAUROSIS | Dependovirus - genetics | Genetic Therapy | Dose-Response Relationship, Immunologic | Immunoglobulin G - blood | Humans | Male | Antibodies, Viral - blood | Immunoenzyme Techniques | CD4-Positive T-Lymphocytes - immunology | Lymphocyte Activation - genetics | Lymphocyte Activation - immunology | Muscle, Skeletal - immunology | Immunoglobulin G - immunology | CD8-Positive T-Lymphocytes - metabolism | Female | Capsid - metabolism | Creatine Kinase - immunology | Muscle, Skeletal - enzymology | Transduction, Genetic | Lipoprotein Lipase - genetics | Transgenes - genetics | CD4-Positive T-Lymphocytes - metabolism | Hyperlipoproteinemia Type I - enzymology | Dependovirus - immunology | Lipoprotein Lipase - biosynthesis | Hyperlipoproteinemia Type I - genetics | Capsid - immunology | B-Lymphocytes - immunology | Creatine Kinase - biosynthesis | Hyperlipoproteinemia Type I - immunology | Interferon-gamma - immunology | Lipoprotein Lipase - immunology | Triglycerides - blood | Antibodies, Viral - immunology | Hyperlipoproteinemia Type I - therapy | CD8-Positive T-Lymphocytes - immunology | Transgenes - immunology | Interferon-gamma - biosynthesis | Gene Therapy
Journal Article
Molecular Therapy - Methods and Clinical Development, ISSN 2329-0501, 04/2015, Volume 2, p. 15029
Adeno-associated virus (AAV) has become one of the most promising vectors in gene transfer in the last 10 years with successful translation to clinical trials... 
MEDICINE, RESEARCH & EXPERIMENTAL
Journal Article
Blood, ISSN 0006-4971, 10/2007, Volume 110, Issue 7, pp. 2334 - 2341
Journal Article
Molecular Therapy, ISSN 1525-0016, 01/2006, Volume 13, Issue 1, pp. 88 - 97
Journal Article
Molecular Therapy, ISSN 1525-0016, 05/2006, Volume 13, Issue S1, pp. S31 - S32
Adeno-associated virus (AAV) vectors hold the potential to treat a variety of inherited and acquired human diseases. One of the confounding issues regarding... 
Journal Article
by Schmidt, Manfred and Verhoeyen, Els and Gobbo, Emilie and Divers, Dominique and Oudrhiri, Noufissa and Griscelli, Frank and Bennaceur-Griscelli, Annelise and Klatzmann, David and Anguela, Xavier M and Sharma, Rajiv and Sharma, Rajiv and Li, Hojun and Li, Hojun and Haurigot, Virginia and Haurigot, Virginia and Haurigot, Virginia and Bhagwat, Anand and Bhagwat, Anand and Davidson, Robert and Davidson, Robert and Zhou, Shangzhen and Zhou, Shangzhen and Zhou, Shangzhen and Doyon, Yannick and Gregory, Philip D and Gregory, Philip D and Gregory, Philip D and Holmes, Michael C and Holmes, Michael C and Holmes, Michael C and High, Katherine A and Carbonaro, Denise and Shaw, Kit and Jin, Xiangyang and Geiger, Sabine and Mishra, Suparna and Cooper, Aaron and DeOliveira, Satiro and Sokolic, Rob and Candotti, Fabio and Carmo, Marlene and Arumugam, Paritha and Alonso-Ferrero, Maria and Schambach, Axel and Schambach, Axel and Baum, Christopher and Baum, Christopher and Baum, Christopher and Risma, Kimberly and Malik, Punam and Jordan, Michael and Rivat, Christine and Booth, Claire and Thrasher, Adrian and Whilding, Lynsey and Archibald, Kyra and Oberg, Daniel and Golan, Talia and Hubert, Ayala and Shemi, Amotz and Khvalevsky, Elina Zorde and Gabai-Malka, Racheli and Focht, Gili and Brunschwig, Zivia and Raskin, Stephen and Goldberg, Nahum and Ben-David, Eli and Peretz, Tamar and Eliakim, Rami and Dankur, Alan and Galun and Rachmur, Itzik and Domb, Avi and Kopelman, Yael and Hantz, Yael and Lahav, Mor and Arbel-Alon, Sagit and Dickson, George and Barkats, Martine and Daboussi, Fayza and Silva, Georges and Cedrone, Frederic and Epinat, Jean Charles and Juillerat, Alexandre and Valton, Julien and Montini, Eugenio and Biffi, Alessandra and Biffi, Alessandra and Calabria, Andrea and Calabria, Andrea and Biasco, Luca and Biasco, Luca and Cesani, Martina and Cesani, Martina and Benedicenti, Fabrizio and Benedicenti, Fabrizio and Plati, Tiziana and Leo, Simone and Zanetti, Gianluigi and Aiuti, Alessandro and ...
Human Gene Therapy, ISSN 1043-0342, 10/2012, Volume 23, Issue 10, pp. A1 - A173
Journal Article
Molecular Therapy, ISSN 1525-0016, 05/2004, Volume 9, Issue S1, pp. S16 - S16
In a Phase I trial for Hemophilia B six subjects underwent infusion of an adeno-associated viral (AAV) vector expressing human Factor IX (F.IX) under the... 
Journal Article
Blood, ISSN 0006-4971, 11/2006, Volume 108, Issue 11, pp. 3269 - 3269
Abstract Use of adeno-associated virus (AAV) to achieve liver-targeted gene transfer for the treatment of hemophilia B in human subjects resulted in the... 
Journal Article
Blood, ISSN 0006-4971, 11/2004, Volume 104, Issue 11, pp. 691 - 691
Abstract Coagulation proteases are crucial for hemostasis and have also been implicated in inflammatory responses, blood vessel formation, and tumor cell... 
Journal Article
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