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Expert Opinion on Biological Therapy Volume 13, 2013 - Issue 1
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Editorial

Alipogene tiparvovec: gene therapy for lipoprotein lipase deficiency

Anthony S WierzbickiGuy's & St. Thomas' Hospitals, Lambeth Palace Road, London SE1 7EH, UK+0207 188 1256; +0207 188 7325; [email protected]
, DM DPhil FRCPath FAHA &
Adie ViljoenLister Hospital, Stevenage, Hertfordshire SG1 4AB, UK
, MBBS FRCPath
Pages 7-10 | Published online: 06 Nov 2012

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Rabia Chaudhry, Adie Viljoen & Anthony S. Wierzbicki. (2018) Pharmacological treatment options for severe hypertriglyceridemia and familial chylomicronemia syndrome. Expert Review of Clinical Pharmacology 11:6, pages 589-598.
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Jooho Lee & Robert A. Hegele. (2017) Investigated treatments for lipoprotein lipase deficiency and related metabolic disorders. Expert Opinion on Orphan Drugs 5:5, pages 411-420.
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Anthony S. Wierzbicki & Adie Viljoen. (2016) Anti-sense oligonucleotide therapies for the treatment of hyperlipidaemia. Expert Opinion on Biological Therapy 16:9, pages 1125-1134.
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Jing Pang, Dick C Chan & Gerald F Watts. (2014) Critical review of non-statin treatments for dyslipoproteinemia. Expert Review of Cardiovascular Therapy 12:3, pages 359-371.
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Toufik Abdul-Rahman, Syed Muhammad Awais Bukhari, Emiliano Cantu Herrera, Wireko Andrew Awuah, Jannel Lawrence, Heloisa de Andrade, Neal Patel, Rohan Shah, Raheel Shaikh, Camilo Andrés Avendaño Capriles, Sebahat Ulusan, Shahzaib Ahmad, Anna Chiara Corriero, Adriana C. Mares, Akshay Goel, Adrija Hajra, Dhrubajyoti Bandyopadhyay & Rahul Gupta. (2022) Lipid Lowering Therapy: An Era Beyond Statins. Current Problems in Cardiology 47:12, pages 101342.
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Nicola Longo & Kent Lai. 2022. Nutrition Management of Inherited Metabolic Diseases. Nutrition Management of Inherited Metabolic Diseases 97 109 .
Subashini C. Thambiah & Leslie Charles Lai. (2021) Diabetic dyslipidaemia. Practical Laboratory Medicine 26, pages e00248.
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Manish Muhuri, Wei Zhan, Yukiko Maeda, Jia Li, Anoushka Lotun, Jennifer Chen, Katelyn Sylvia, Ishani Dasgupta, Motahareh Arjomandnejad, Thomas Nixon, Allison M. Keeler, Sangeetha Manokaran, Ran He, Qin Su, Phillip W. L. Tai & Guangping Gao. (2021) Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction. Frontiers in Immunology 12.
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Sanjay Kumar, Anchal Singhal, Uma Narang, Sweta Mishra & Pratibha Kumari. (2020) Recent Progresses in Organic-Inorganic Nano Technological Platforms for Cancer Therapeutics. Current Medicinal Chemistry 27:35, pages 6015-6056.
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Gabrielle Barbosa Pinto, Flávia Maria Lins Mendes & Adelaide Maria de Souza Antunes. (2020) Technological Profile of Lipases in the Pharmaceutical Industry. Mini-Reviews in Organic Chemistry 17:6, pages 701-716.
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Balaji Balakrishnan & Ernest David. (2019) Biopolymers augment viral vectors based gene delivery. Journal of Biosciences 44:4.
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Yuanyuan Xiao, Manish Muhuri, Shaoyong Li, Wanru Qin, Guangchao Xu, Li Luo, Jia Li, Alexander J. Letizia, Sean K. Wang, Ying Kai Chan, Chunmei Wang, Sebastian P. Fuchs, Dan Wang, Qin Su, M. Abu Nahid, George M. Church, Michael Farzan, Li Yang, Yuquan Wei, Ronald C. Desrosiers, Christian Mueller, Phillip W.L. Tai & Guangping Gao. (2019) Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity. JCI Insight 4:13.
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Margherita Botta, Elisabeth Maurer, Massimiliano Ruscica, Stefano Romeo, Thomas M. Stulnig & Piero Pingitore. (2019) Deciphering the role of V200A and N291S mutations leading to LPL deficiency. Atherosclerosis 282, pages 45-51.
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Alfred Adiamah, Emmanouil Psaltis, Martin Crook & Dileep N. Lobo. (2018) A systematic review of the epidemiology, pathophysiology and current management of hyperlipidaemic pancreatitis. Clinical Nutrition 37:6, pages 1810-1822.
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Ursula Kassner, Tim Hollstein, Thomas Grenkowitz, Marion Wühle-Demuth, Bastian Salewsky, Ilja Demuth, Michaela DippelElisabeth Steinhagen-Thiessen. (2018) Gene Therapy in Lipoprotein Lipase Deficiency: Case Report on the First Patient Treated with Alipogene Tiparvovec Under Daily Practice Conditions. Human Gene Therapy 29:4, pages 520-527.
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Anthony S. Wierzbicki. 2018. Endocrine and Metabolic Medical Emergencies. Endocrine and Metabolic Medical Emergencies 742 759 .
Masayuki Kuroda, Yasushi Saito, Masayuki AsoKoutaro Yokote. (2018) A Novel Approach to the Treatment of Plasma Protein Deficiency: <i>Ex Vivo</i>-Manipulated Adipocytes for Sustained Secretion of Therapeutic Proteins. Chemical and Pharmaceutical Bulletin 66:3, pages 217-224.
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Sasi Neelamekam, See Kwok, Rachel Malone, Anthony S. Wierzbicki & Handrean Soran. (2017) The impact of lipoprotein lipase deficiency on health-related quality of life: a detailed, structured, qualitative study. Orphanet Journal of Rare Diseases 12:1.
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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.
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Adie Viljoen, Supriya Joshi & Anthony S. Wierzbicki. 2017. Textbook of Diabetes. Textbook of Diabetes 643 652 .
Tetsuya Ishii. (2017) Germ line genome editing in clinics: the approaches, objectives and global society. Briefings in Functional Genomics 16:1, pages 46-56.
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Sophie Carter & Yannick Doyon. 2017. Hereditary Tyrosinemia. Hereditary Tyrosinemia 231 243 .
Yu-Shan Tseng, Kim Van Vliet, Lavanya Rao, Robert McKenna, Barry J. Byrne, Aravind Asokan & Mavis Agbandje-McKenna. (2016) Generation and characterization of anti-Adeno-associated virus serotype 8 (AAV8) and anti-AAV9 monoclonal antibodies. Journal of Virological Methods 236, pages 105-110.
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A. A. Momin, M. P. Bankar & G. M. Bhoite. (2015) Study of Common Genetic Variant S447X in Lipoprotein Lipase and Its Association with Lipids and Lipoproteins in Type 2 Diabetic Patients. Indian Journal of Clinical Biochemistry 31:3, pages 286-293.
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O. A. Bezborodova, E. R. Nemtsova, R. I. Yakubovskaya & A. D. Kaprin. (2016) Gene therapy is a new area in medicine. Onkologiya. Zhurnal imeni P.A.Gertsena 5:2, pages 64.
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Uma Ramaswami & Steve Humphries. 2016. Inborn Metabolic Diseases. Inborn Metabolic Diseases 441 454 .
Amanda J. Brahm & Robert A. Hegele. (2015) Chylomicronaemia—current diagnosis and future therapies. Nature Reviews Endocrinology 11:6, pages 352-362.
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M Ferrand, A Galy & F Boisgerault. (2014) A dystrophic muscle broadens the contribution and activation of immune cells reacting to rAAV gene transfer. Gene Therapy 21:9, pages 828-839.
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Claudia Gonzaga-Jauregui, Sabina Mir, Samantha Penney, Shalini Jhangiani, Craig Midgen, Milton Finegold, Donna M. Muzny, Min Wang, Carlos A. Bacino, Richard A. Gibbs, James R. Lupski, Richard Kellermayer & Neil A. Hanchard. (2014) Whole-Exome Sequencing Reveals GPIHBP1 Mutations in Infantile Colitis With Severe Hypertriglyceridemia. Journal of Pediatric Gastroenterology & Nutrition 59:1, pages 17-21.
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Jordi Pérez-López. (2014) Terapia génica en el tratamiento de los errores congénitos del metabolismo. Medicina Clínica 142:12, pages 549-553.
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Victoria J. McCurdyAime K. JohnsonHeather L. Gray-EdwardsAshley N. RandleBrandon L. BrunsonNancy E. MorrisonNouha SalibiJacob A. JohnsonMisako HwangRonald J. BeyersStanley G. LeroyStacy MaitlandThomas S. DenneyNancy R. CoxHenry J. BakerMiguel Sena-EstevesDouglas R. Martin. (2014) Sustained Normalization of Neurological Disease after Intracranial Gene Therapy in a Feline Model. Science Translational Medicine 6:231.
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Míriam Badia-Villanueva, Pere Carulla, Montserrat Carrascal, Joaquín Abián, Miquel Llobera, Albert Casanovas & M. Dolores López-Tejero. (2014) Lipoprotein lipase isoelectric point isoforms in humans. Biochemical and Biophysical Research Communications 445:2, pages 480-485.
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Maximilian H?rner, Beate Kaufmann, Gabriella Cotugno, Ellen Wiedtke, Hildegard B?ning, Dirk Grimm & Wilfried Weber. (2014) A chemical switch for controlling viral infectivity. Chem. Commun. 50:71, pages 10319-10322.
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Wilbert S AronowAnthony S Wierzbicki. 2013. Advances in Dyslipidemia. Advances in Dyslipidemia 58 69 .
Gerald F. Watts, Esther M. M. Ooi & Dick C. Chan. (2013) Demystifying the management of hypertriglyceridaemia. Nature Reviews Cardiology 10:11, pages 648-661.
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Mfon Ewang-Emukowhate & Anthony S. Wierzbicki. (2013) Lipid-Lowering Agents. Journal of Cardiovascular Pharmacology and Therapeutics 18:5, pages 401-411.
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