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Safety profile of recombinant adeno-associated viral vectors: focus on alipogene tiparvovec (Glybera®)

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Jin Ding & Wei-Fen Xie. 2018. Stem Cells and Cancer in Hepatology. Stem Cells and Cancer in Hepatology 289 315 .
Kjetil Retterstøl, Ingunn Narverud, Randi Selmer, Knut E. Berge, Ingvild V. Osnes, Stine M. Ulven, Bente Halvorsen, Pål Aukrust, Kirsten B. Holven & Per O. Iversen. (2017) Severe hypertriglyceridemia in Norway: prevalence, clinical and genetic characteristics. Lipids in Health and Disease 16:1.
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Crossref
Michael F. Naso, Brian Tomkowicz, William L. PerryIIIIII & William R. Strohl. (2017) Adeno-Associated Virus (AAV) as a Vector for Gene Therapy. BioDrugs 31:4, pages 317-334.
Crossref
Elisabeth Steinhagen-Thiessen, Erik Stroes, Handrean Soran, Colin Johnson, Philippe Moulin, Giorgio Iotti, Marco Zibellini, Bas Ossenkoppele, Michaela Dippel & Maurizio R. Averna. (2017) The role of registries in rare genetic lipid disorders: Review and introduction of the first global registry in lipoprotein lipase deficiency. Atherosclerosis 262, pages 146-153.
Crossref
William F Goins, Bonnie Hall, Justus B Cohen & Joseph C Glorioso. (2016) Retargeting of herpes simplex virus (HSV) vectors. Current Opinion in Virology 21, pages 93-101.
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Thomas Michler, Stefanie Große, Stefan Mockenhaupt, Natalie Röder, Ferdinand Stückler, Bettina Knapp, Chunkyu Ko, Mathias Heikenwalder, Ulrike Protzer & Dirk Grimm. (2016) Blocking sense‐strand activity improves potency, safety and specificity of anti‐hepatitis B virus short hairpin RNA . EMBO Molecular Medicine 8:9, pages 1082-1098.
Crossref
Zhuo Cheng, Xiaofeng Li & Jin Ding. (2016) Characteristics of liver cancer stem cells and clinical correlations. Cancer Letters 379:2, pages 230-238.
Crossref
Handrean Soran, Jonathan D. Schofield, Safwaan Adam & Paul N. Durrington. (2016) Diabetic dyslipidaemia. Current Opinion in Lipidology 27:4, pages 313-322.
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Franziska Hentzschel, Anne-Kathrin Herrmann, Ann-Kristin Mueller & Dirk Grimm. (2016) Plasmodium meets AAV-the (un)likely marriage of parasitology and virology, and how to make the match . FEBS Letters 590:13, pages 2027-2045.
Crossref
Otto-Wilhelm Merten & Bruno Gaillet. (2016) Viral vectors for gene therapy and gene modification approaches. Biochemical Engineering Journal 108, pages 98-115.
Crossref
Young-Dong Kim, Prasad Pofali, Tae-Eun Park, Bijay Singh, Kihyun Cho, Sushila Maharjan, Prajakta Dandekar, Ratnesh Jain, Yun-Jaie Choi, Rohidas Arote & Chong-Su Cho. (2016) Gene therapy for bone tissue engineering. Tissue Engineering and Regenerative Medicine 13:2, pages 111-125.
Crossref
Yi-Gang Wang. (2016) Targeting adeno-associated virus and adenoviral gene therapy for hepatocellular carcinoma. World Journal of Gastroenterology 22:1, pages 326.
Crossref
Matheus HW Crommentuijn, Rami Kantar, David P Noske, W Peter Vandertop, Christian E Badr, Thomas Würdinger, Casey A Maguire & Bakhos A Tannous. (2016) Systemically administered AAV9-sTRAIL combats invasive glioblastoma in a patient-derived orthotopic xenograft model. Molecular Therapy - Oncolytics 3.
Crossref
Pierre-Olivier Buclez, Gabriella Dias Florencio, Karima Relizani, Cyriaque Beley, Luis Garcia & Rachid Benchaouir. (2016) Rapid, scalable, and low-cost purification of recombinant adeno-associated virus produced by baculovirus expression vector system. Molecular Therapy - Methods & Clinical Development 3, pages 16035.
Crossref
Otto-Wilhelm Merten, Matthias Hebben & Chiara Bovolenta. (2016) Production of lentiviral vectors. Molecular Therapy - Methods & Clinical Development 3, pages 16017.
Crossref
John G. Hardy. 2016. Engineering of Nanobiomaterials. Engineering of Nanobiomaterials 119 144 .
Daniel Gaudet & Diane Brisson. (2015) Gene-based therapies in lipidology. Current Opinion in Lipidology 26:6, pages 553-565.
Crossref
Sujata Halder, Kim Van Vliet, J. Kennon Smith, Thao Thi Phuong Duong, Robert McKenna, James M. Wilson & Mavis Agbandje-McKenna. (2015) Structure of neurotropic adeno-associated virus AAVrh.8. Journal of Structural Biology 192:1, pages 21-36.
Crossref
David PépinAmanda SosulskiLihua ZhangDan WangVinod Vathipadiekal, Katherine HendrenCaroline M. ColettiAaron YuCesar M. Castro, Michael J. Birrer, Guangping GaoPatricia K. Donahoe. (2015) AAV9 delivering a modified human Mullerian inhibiting substance as a gene therapy in patient-derived xenografts of ovarian cancer. Proceedings of the National Academy of Sciences 112:32.
Crossref
Hanna P. Lesch, Kati M. Heikkilä, Eevi M. Lipponen, Piia Valonen, Achim Müller, Eva Räsänen, Tarja Tuunanen, Minna M. Hassinen, Nigel Parker, Minna Karhinen, Robert Shaw & Seppo Ylä-Herttuala. (2015) Process Development of Adenoviral Vector Production in Fixed Bed Bioreactor: From Bench to Commercial Scale. Human Gene Therapy 26:8, pages 560-571.
Crossref
Mara Martín-AlonsoAna B. García-RedondoDongchuan GuoEmilio CamafeitaFernando MartínezArántzazu AlfrancaNerea Méndez-BarberoÁngela PollánCristina Sánchez-CamachoDavid T. DenhardtMotoharu SeikiJesús VázquezMercedes SalaicesJuan Miguel RedondoDianna MilewiczAlicia G. Arroyo. (2015) Deficiency of MMP17/MT4-MMP Proteolytic Activity Predisposes to Aortic Aneurysm in Mice. Circulation Research 117:2.
Crossref
Weihong Qu, Mingxi Wang, Yaqing Wu, Yinghui Lv, Qizhao Wang & Ruian Xu. (2015) Calcium-ion-modulated ceramic hydroxyapatite resin for the scalable purification of recombinant Adeno-Associated Virus serotype 9. Journal of Chromatography B 990, pages 15-22.
Crossref
Piergiuseppe Nestola, Cristina Peixoto, Ricardo R. J. S Silva, Paula M. Alves, José P. B. Mota & Manuel J. T. Carrondo. (2015) Improved virus purification processes for vaccines and gene therapy. Biotechnology and Bioengineering 112:5, pages 843-857.
Crossref
Rachael S. Felberbaum. (2015) The baculovirus expression vector system: A commercial manufacturing platform for viral vaccines and gene therapy vectors. Biotechnology Journal 10:5, pages 702-714.
Crossref
Xiaohui Zhang, Subrata K Das, Samuel F Passi, Hironori Uehara, Austin Bohner, Marcus Chen, Michelle Tiem, Bonnie Archer & Balamurali K Ambati. (2015) AAV2 Delivery of Flt23k Intraceptors Inhibits Murine Choroidal Neovascularization. Molecular Therapy 23:2, pages 226-234.
Crossref
Lesley J. Scott. (2015) Alipogene Tiparvovec: A Review of Its Use in Adults with Familial Lipoprotein Lipase Deficiency. Drugs 75:2, pages 175-182.
Crossref
Andreas Plückthun. (2015) Designed Ankyrin Repeat Proteins (DARPins): Binding Proteins for Research, Diagnostics, and Therapy. Annual Review of Pharmacology and Toxicology 55:1, pages 489-511.
Crossref
Gabriella Dias Florencio, Guillaume Precigout, Cyriaque Beley, Pierre-Olivier Buclez, Luis Garcia & Rachid Benchaouir. (2015) Simple downstream process based on detergent treatment improves yield and in vivo transduction efficacy of adeno-associated virus vectors. Molecular Therapy - Methods & Clinical Development 2, pages 15024.
Crossref
Bas Blits, Deniz Kirik, Harald Petry & Stephan Hermening. 2015. Gene Delivery and Therapy for Neurological Disorders. Gene Delivery and Therapy for Neurological Disorders 67 83 .
Ruth Röder & Ernst Wagner. (2014) Sequence-defined shuttles for targeted nucleic acid and protein delivery. Therapeutic Delivery 5:9, pages 1025-1045.
Crossref
. (2014) Augmenting PBGD Expression in the Liver as a Novel Gene Therapy for Acute Intermittent Porphyria (AIPgene). Human Gene Therapy Clinical Development 25:2, pages 61-63.
Crossref
Mukerrem Hale Tasyurek, Hasan Ali Altunbas, Halit Canatan, Thomas S. Griffith & Salih Sanlioglu. (2014) GLP-1-mediated gene therapy approaches for diabetes treatment. Expert Reviews in Molecular Medicine 16.
Crossref
Milton W. TaylorMilton W. Taylor. 2014. Viruses and Man: A History of Interactions. Viruses and Man: A History of Interactions 337 354 .

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