Effect of Alipogene Tiparvovec (AAV1-LPL S447X ) on Postprandial Chylomicron Metabolism in Lipoprotein Lipase-Deficient Patients

INFLUENCES OF GLUCOSE LOADING AND OF INJECTED INSULIN ON HEPATIC GLUCOSE OUTPUT*

Steele, R. D. (1959) · Annals of the New York Academy of Sciences · doi:10.1111/j.1749-6632.1959.tb44923.x · Cited by 1,680 · see citing works

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

Brantly, M. L., Chulay, J. D., Wang, L., Mueller, C., Humphries, M., Spencer, L. T., Rouhani, F., Conlon, T. J., Calcedo, R., Betts, M. R., et al. (2009) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0904514106 · Cited by 327 · see citing works

Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer

Rivera, V. M., Gao, G., Grant, R. L., Schnell, M. A., Zoltick, P. W., Rozamus, L. W., Clackson, T., & Wilson, J. M. (2004) · Blood · doi:10.1182/blood-2004-06-2501 · Cited by 272 · see citing works

Preferential Uptake of Dietary Fatty Acids in Adipose Tissue and Muscle in the Postprandial Period

Bickerton, A. S. T., Roberts, R., Fielding, B. A., Hodson, L., Blaak, E. E., Wagenmakers, A. J. M., Gilbert, M., Karpe, F., & Frayn, K. N. (2006) · Diabetes · doi:10.2337/db06-0822 · Cited by 247 · see citing works

Lipoprotein lipase and the disposition of dietary fatty acids

Fielding, B. A., & Frayn, K. N. (1998) · British Journal Of Nutrition · doi:10.1017/s0007114598001585 · Cited by 231 · see citing works

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(2001) · Medical Entomology and Zoology · Cited by 227 · see citing works

Intramuscular Administration of AAV1-Lipoprotein Lipase ^S447X Lowers Triglycerides in Lipoprotein Lipase–Deficient Patients

Stroes, E. S., Nierman, M. C., Meulenberg, J. J., Franssen, R., Twisk, J., Henny, C. P., Maas, M. M., Zwinderman, A. H., Ross, C., Aronica, E., et al. (2008) · Arteriosclerosis, Thrombosis, and Vascular Biology · doi:10.1161/atvbaha.108.175620 · Cited by 209 · see citing works

Evidence of Multiyear Factor IX Expression by AAV-Mediated Gene Transfer to Skeletal Muscle in an Individual with Severe Hemophilia B

Jiang, H., Pierce, G. F., Ozelo, M. C., Paula, E. V. D., Vargas, J. A., Smith, P., Sommer, J. M., Luk, A. Y., Manno, C. S., High, K. A., et al. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.05.004 · Cited by 207 · see citing works

Lipid-induced pancreatic β-cell dysfunction: focus on in vivo studies

Giacca, A., Xiao, C., Oprescu, A. I., Carpentier, A. C., & Lewis, G. F. (2010) · American Journal of Physiology-Endocrinology and Metabolism · doi:10.1152/ajpendo.00416.2010 · Cited by 196 · see citing works

Lipoprotein lipase: physiology, biochemistry, and molecular biology

Ira, J. G. (2001) · Frontiers in bioscience · doi:10.2741/goldberg · Cited by 195 · see citing works

Regulation of fatty acid movement in human adipose tissue in the postabsorptive-to-postprandial transition

Frayn, K. N., Shadid, S., Hamlani, R., Humphreys, S. M., Clark, M. L., Fielding, B. A., Boland, O., & Coppack, S. W. (1994) · American Journal of Physiology-Endocrinology and Metabolism · doi:10.1152/ajpendo.1994.266.3.e308 · Cited by 188 · see citing works

Postprandial Free Fatty Acid Kinetics Are Abnormal in Upper Body Obesity

Roust, L. R., & Jensen, M. D. (1993) · Diabetes · doi:10.2337/diab.42.11.1567 · Cited by 164 · see citing works

Regulation of Dietary Fatty Acid Entrapment in Subcutaneous Adipose Tissue and Skeletal Muscle

Evans, K., Burdge, G. C., Wootton, S. A., Clark, M. L., & Frayn, K. N. (2002) · Diabetes · doi:10.2337/diabetes.51.9.2684 · Cited by 161 · see citing works

Lipoprotein Lipase S447X: A Naturally Occurring Gain-of-Function Mutation

Rip, J., Nierman, M. C., Ross, C. J., Jukema, J. W., Hayden, M. R., Kastelein, J. J. P., Stroes, E. S. G., & Kuivenhoven, J. A. (2006) · Arteriosclerosis, Thrombosis, and Vascular Biology · doi:10.1161/01.atv.0000219283.10832.43 · Cited by 157 · see citing works

Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model

Arruda, V. R., Stedman, H. H., Nichols, T. C., Haskins, M. E., Nicholson, M., Herzog, R. W., Couto, L. B., & High, K. A. (2004) · Blood · doi:10.1182/blood-2004-07-2908 · Cited by 153 · see citing works

Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency

Gaudet, D., de Wal, J., Tremblay, K., Déry, S., van Deventer, S. J. H., Freidig, A. P., Brisson, D., & Méthot, J. (2010) · Atherosclerosis Supplements · doi:10.1016/j.atherosclerosissup.2010.03.004 · Cited by 122 · see citing works

Systemic and Forearm Triglyceride Metabolism

Miles, J. M., Park, Y., Walewicz, D., Russell-Lopez, C., Windsor, S. L., Isley, W. L., Coppack, S. W., & Harris, W. S. (2004) · Diabetes · doi:10.2337/diabetes.53.3.521 · Cited by 114 · see citing works

What Does the Measurement of Whole-Body Fatty Acid Rate of Appearance in Plasma by Using a Fatty Acid Tracer Really Mean?

Mittendorfer, B., Liem, O., Patterson, B. W., Miles, J. M., & Klein, S. (2003) · Diabetes · doi:10.2337/diabetes.52.7.1641 · Cited by 114 · see citing works

Nocturnal and Postprandial Free Fatty Acid Kinetics in Normal and Type 2 Diabetic Subjects

Miles, J. M., Wooldridge, D., Grellner, W. J., Windsor, S. L., Isley, W. L., Klein, S., & Harris, W. S. (2003) · Diabetes · doi:10.2337/diabetes.52.3.675 · Cited by 103 · see citing works

Uptake of individual fatty acids into adipose tissue in relation to their presence in the diet

Summers, L. K. M., Barnes, S. C., Fielding, B. A., Beysen, C., Ilic, V., Humphreys, S. M., & Frayn, K. N. (2000) · American Journal of Clinical Nutrition · doi:10.1093/ajcn/71.6.1470 · Cited by 89 · see citing works

Correction of Feline Lipoprotein Lipase Deficiency with Adeno-Associated Virus Serotype 1-Mediated Gene Transfer of the Lipoprotein Lipase S447X Beneficial Mutation

Ross, C. J., Twisk, J., Bakker, A. C., Miao, F., Verbart, D. J., Rip, J., Godbey, T., Dijkhuizen, P. A., Hermens, W. T. J. M. C., Kastelein, J. J. P., et al. (2006) · Human Gene Therapy · doi:10.1089/hum.2006.17.487 · Cited by 82 · see citing works

Postprandial fatty acid metabolism in the development of lipotoxicity and type 2 diabetes

Carpentier, A. C. (2008) · Diabetes & Metabolism · doi:10.1016/j.diabet.2007.10.009 · Cited by 79 · see citing works

Long-Term Correction of Murine Lipoprotein Lipase Deficiency with AAV1-Mediated Gene Transfer of the Naturally Occurring LPL S447X Beneficial Mutation

Ross, C. J., Twisk, J., Meulenberg, J. J. M., Liu, G., van den Oever, K., Moraal, E., Hermens, W. T., Rip, J., Kastelein, J. J. P., Kuivenhoven, J. A., et al. (2004) · Human Gene Therapy · doi:10.1089/hum.2004.15.906 · Cited by 78 · see citing works

Assessment of methods for improving tracer estimation of non-steady-state rate of appearance

Gastaldelli, A., Coggan, A. R., & Wolfe, R. R. (1999) · Journal of Applied Physiology · doi:10.1152/jappl.1999.87.5.1813 · Cited by 69 · see citing works

On the suppression of plasma nonesterified fatty acids by insulin during enhanced intravascular lipolysis in humans

Carpentier, A. C., Frisch, F., Cyr, D., Généreux, P., Patterson, B. W., Giguère, R., & Baillargeon, J. (2005) · American Journal of Physiology-Endocrinology and Metabolism · doi:10.1152/ajpendo.00073.2005 · Cited by 69 · see citing works

Gene Therapy for Lipoprotein Lipase Deficiency: Working Toward Clinical Application

Rip, J., Nierman, M. C., Sierts, J. A., Petersen, W., van den Oever, K., van Raalte, D. H., Ross, C. J., Hayden, M. R., Bakker, A. C., Dijkhuizen, P. A., et al. (2005) · Human Gene Therapy · doi:10.1089/hum.2005.16.1276 · Cited by 63 · see citing works

Impaired Plasma Nonesterified Fatty Acid Tolerance Is an Early Defect in the Natural History of Type 2 Diabetes

Brassard, P., Frisch, F., Lavoie, F., Cyr, D., Bourbonnais, A., Cunnane, S. C., Patterson, B. W., Drouin, R., Baillargeon, J., & Carpentier, A. C. (2008) · The Journal of Clinical Endocrinology & Metabolism · doi:10.1210/jc.2007-1670 · Cited by 54 · see citing works

Alipogene tiparvovec, an adeno-associated virus encoding the Ser(447)X variant of the human lipoprotein lipase gene for the treatment of patients with lipoprotein lipase deficiency.

Burnett, J. R., & Hooper, A. J. (2009) · PubMed · Cited by 52 · see citing works

Measurement of non-steady-state free fatty acid turnover

Jensen, M. D., Heiling, V. J., & Miles, J. J. (1990) · American Journal of Physiology-Endocrinology and Metabolism · doi:10.1152/ajpendo.1990.258.1.e103 · Cited by 51 · see citing works

Chylomicron-retinyl palmitate clearance in type I hyperlipidemic families.

Sprecher, D. L., Knauer, S. L., Black, D. M., Kaplan, L. A., Akeson, A. A., Dusing, M. R., Lattier, D. L., Stein, E. A., Rymaszewski, M., & Wiginton, D. A. (1991) · Journal of Clinical Investigation · doi:10.1172/jci115402 · Cited by 49 · see citing works

Increased Postprandial Nonesterified Fatty Acid Appearance and Oxidation in Type 2 Diabetes Is Not Fully Established in Offspring of Diabetic Subjects

Normand-Lauzière, F., Frisch, F., Labbé, S. M., Bhérer, P., Gagnon, R., Cunnane, S. C., & Carpentier, A. C. (2010) · PLoS ONE · doi:10.1371/journal.pone.0010956 · Cited by 49 · see citing works

Enhanced escape of non-esterified fatty acids from tissue uptake: its role in impaired insulin-induced lowering of total rate of appearance in obesity and Type II diabetes mellitus

Riemens, S., Sluiter, W., & Dullaart, R. (2000) · Diabetologia · doi:10.1007/s001250051324 · Cited by 40 · see citing works

A new method for the study of chylomicron kinetics in vivo

Park, Y., Grellner, W. J., Harris, W. S., & Miles, J. M. (2000) · American Journal of Physiology-Endocrinology and Metabolism · doi:10.1152/ajpendo.2000.279.6.e1258 · Cited by 34 · see citing works

Mechanism of insulin-stimulated clearance of plasma nonesterified fatty acids in humans

Carpentier, A. C., Frisch, F., Brassard, P., Lavoie, F., Bourbonnais, A., Cyr, D., Giguère, R., & Baillargeon, J. (2006) · American Journal of Physiology-Endocrinology and Metabolism · doi:10.1152/ajpendo.00423.2006 · Cited by 27 · see citing works

Relationship between Total and High Molecular Weight Adiponectin Levels and Plasma Nonesterified Fatty Acid Tolerance during Enhanced Intravascular Triacylglycerol Lipolysis in Men

Lavoie, F., Frisch, F., Brassard, P., Normand-Lauzière, F., Cyr, D., Gagnon, R., Drouin, R., Baillargeon, J., & Carpentier, A. C. (2008) · The Journal of Clinical Endocrinology & Metabolism · doi:10.1210/jc.2008-1021 · Cited by 21 · see citing works

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

Mingozzi, F., & High, K. A. (2013) · Blood · doi:10.1182/blood-2013-01-306647 · Cited by 955 · see citing works

Engineering adeno-associated viruses for clinical gene therapy

Kotterman, M. A., & Schaffer, D. V. (2014) · Nature Reviews Genetics · doi:10.1038/nrg3742 · Cited by 761 · see citing works

Adverse Health Consequences of Performance-Enhancing Drugs: An Endocrine Society Scientific Statement

Pope, H. G., Wood, R. I., Rogol, A. D., Nyberg, F., Bowers, L. D., & Bhasin, S. (2013) · Endocrine Reviews · doi:10.1210/er.2013-1058 · Cited by 680 · see citing works

AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer

Costa Verdera, H., Kuranda, K., & Mingozzi, F. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2019.12.010 · Cited by 641 · see citing works

Clinical review on triglycerides

Laufs, U., Parhofer, K. G., Ginsberg, H. N., & Hegele, R. A. (2020) · European Heart Journal · doi:10.1093/eurheartj/ehz785 · Cited by 544 · see citing works

Viral Vectors for Gene Therapy: Translational and Clinical Outlook

Kotterman, M. A., Chalberg, T. W., & Schaffer, D. V. (2015) · Annual Review of Biomedical Engineering · doi:10.1146/annurev-bioeng-071813-104938 · Cited by 444 · see citing works

Lipoproteins and lipids in cardiovascular disease: from mechanistic insights to therapeutic targeting

Soppert, J., Lehrke, M., Marx, N., Jankowski, J., & Noels, H. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.07.019 · Cited by 419 · see citing works

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

Gaudet, D., Méthot, J., Déry, S., Brisson, D., Essiembre, C., Tremblay, G., Tremblay, K., de Wal, J., Twisk, J., van den Bulk, N., et al. (2013) · Gene Therapy · doi:10.1038/gt.2012.43 · Cited by 404 · see citing works

Chylomicronaemia–current diagnosis and future therapies

Brahm, A. J., & Hegele, R. A. (2015) · Nature Reviews Endocrinology · doi:10.1038/nrendo.2015.26 · Cited by 356 · see citing works

Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

Keeler, A. M., & Flotte, T. R. (2019) · Annual Review of Virology · doi:10.1146/annurev-virology-092818-015530 · Cited by 322 · see citing works

Genetics and causality of triglyceride-rich lipoproteins in atherosclerotic cardiovascular disease

Rosenson, R. S., Davidson, M. H., Hirsh, B. J., Kathiresan, S., & Gaudet, D. (2014) · Journal of the American College of Cardiology · doi:10.1016/j.jacc.2014.09.042 · Cited by 260 · see citing works

Unraveling the complex story of immune responses to AAV vectors trial after trial.

Vandamme, C., Adjali, O., & Mingozzi, F. (2017) · Human Gene Therapy · doi:10.1089/hum.2017.150 · Cited by 234 · see citing works

ATP-citrate lyase (ACLY) in lipid metabolism and atherosclerosis: An updated review

Feng, X., Zhang, L., Xu, S., & Shen, A.-Z. (2019) · Progress in Lipid Research · doi:10.1016/j.plipres.2019.101006 · Cited by 233 · see citing works

Adeno-associated virus (AAV) vectors in cancer gene therapy

Santiago-Ortiz, J. L., & Schaffer, D. V. (2016) · Journal of Controlled Release · doi:10.1016/j.jconrel.2016.01.001 · Cited by 231 · see citing works

A largely random AAV integration profile after LPLD gene therapy

Kaeppel, C., Beattie, S. G., Fronza, R., van Logtenstein, R., Salmon, F., Schmidt, S., Wolf, S., Nowrouzi, A., Glimm, H., Kalle, C. V., et al. (2013) · Nature Medicine · doi:10.1038/nm.3230 · Cited by 190 · see citing works

Lessons learned from the clinical development and market authorization of Glybera

Bryant, L. M., Christopher, D. M., Giles, A. R., Hinderer, C., Rodriguez, J. L., Smith, J. B., Traxler, E. A., Tycko, J., Wojno, A. P., & Wilson, J. M. (2013) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2013.087 · Cited by 189 · see citing works

Postprandial hypertriglyceridemia as a coronary risk factor

Borén, J., Matikainen, N., Adiels, M., & Taskinen, M.-R. (2014) · Clinica Chimica Acta · doi:10.1016/j.cca.2014.01.015 · Cited by 182 · see citing works

In vivo genome editing improves motor function and extends survival in a mouse model of ALS

Gaj, T., Ojala, D. S., Ekman, F. K., Byrne, L. C., Limsirichai, P., & Schaffer, D. V. (2017) · Science Advances · doi:10.1126/sciadv.aar3952 · Cited by 185 · see citing works

Gene therapy for lipoprotein lipase deficiency

Gaudet, D., Méthot, J., & Kastelein, J. (2012) · Current Opinion in Lipidology · doi:10.1097/mol.0b013e3283555a7e · Cited by 178 · see citing works

Antibody neutralization poses a barrier to intravitreal adeno-associated viral vector gene delivery to non-human primates

Kotterman, M. A., Yin, L., Strazzeri, J. M., Flannery, J. G., Merigan, W. H., & Schaffer, D. V. (2015) · Gene Therapy · doi:10.1038/gt.2014.115 · Cited by 169 · see citing works

Lipid-Lowering Agents: Targets Beyond PCSK9

Hegele, R. A., & Tsimikas, S. (2019) · Circulation Research · doi:10.1161/circresaha.118.313171 · Cited by 171 · see citing works

Recent advances in pharmacotherapy for hypertriglyceridemia

Sahebkar, A., Chew, G. T., & Watts, G. F. (2014) · Progress in Lipid Research · doi:10.1016/j.plipres.2014.07.002 · Cited by 166 · see citing works

Cardiovascular Gene Therapy: Past, Present, and Future

Ylä‐Herttuala, S., & Baker, A. H. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.03.027 · Cited by 166 · see citing works

CRISPR-Cas9-Mediated Genome Editing Increases Lifespan and Improves Motor Deficits in a Huntington’s Disease Mouse Model

Ekman, F. K., Ojala, D. S., Adil, M. M., Zapana, P. A. L., Schaffer, D. V., & Gaj, T. (2019) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2019.07.009 · Cited by 163 · see citing works

Immune Responses to Intramuscular Administration of Alipogene Tiparvovec (AAV1-LPL ^S447X ) in a Phase II Clinical Trial of Lipoprotein Lipase Deficiency Gene Therapy

Ferreira, V., Twisk, J., Kwikkers, K., Aronica, E., Brisson, D., Methot, J., Petry, H., & Gaudet, D. (2014) · Human Gene Therapy · doi:10.1089/hum.2013.169 · Cited by 137 · see citing works

Mapping the AAV Capsid Host Antibody Response toward the Development of Second Generation Gene Delivery Vectors

Tseng, Y.-S., & Agbandje‐McKenna, M. (2014) · Frontiers in Immunology · doi:10.3389/fimmu.2014.00009 · Cited by 134 · see citing works

The Chylomicronemia Syndrome Is Most Often Multifactorial: A Narrative Review of Causes and Treatment

Chait, A., & Eckel, R. H. (2019) · Annals of Internal Medicine · doi:10.7326/m19-0203 · Cited by 128 · see citing works

Alipogene Tiparvovec: A Review of Its Use in Adults with Familial Lipoprotein Lipase Deficiency

Scott, L. J. (2015) · Drugs · doi:10.1007/s40265-014-0339-9 · Cited by 130 · see citing works

Immune responses to AAV-vectors, the Glybera example from bench to bedside

Ferreira, V., Petry, H., & Salmon, F. (2014) · Frontiers in Immunology · doi:10.3389/fimmu.2014.00082 · Cited by 120 · see citing works

Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications

Gaj, T., Epstein, B. E., & Schaffer, D. V. (2015) · Molecular Therapy · doi:10.1038/mt.2015.151 · Cited by 117 · see citing works

Adeno-Associated Viral Vectors in Neuroscience Research

Haggerty, D. L., Grecco, G. G., Reeves, K. C., & Atwood, B. K. (2019) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2019.11.012 · Cited by 123 · see citing works

An Alternate Route for Adeno-associated Virus (AAV) Entry Independent of AAV Receptor

Dudek, A. M., Pillay, S., Puschnik, A. S., Nagamine, C. M., Cheng, F., Qiu, J., Carette, J. E., & Vandenberghe, L. H. (2018) · Journal of Virology · doi:10.1128/jvi.02213-17 · Cited by 116 · see citing works

Gene therapy enters the pharma market: The short story of a long journey

Büning, H. (2013) · EMBO Molecular Medicine · doi:10.1002/emmm.201202291 · Cited by 113 · see citing works

Effect of the DGAT1 inhibitor pradigastat on triglyceride and apoB48 levels in patients with familial chylomicronemia syndrome

Meyers, C. D., Tremblay, K., Amer, A., Chen, J., Jiang, L., & Gaudet, D. (2015) · Lipids in Health and Disease · doi:10.1186/s12944-015-0006-5 · Cited by 110 · see citing works

Postprandial lipemia as a cardiometabolic risk factor

Pirillo, A., Norata, G. D., & Catapano, A. L. (2014) · Current Medical Research and Opinion · doi:10.1185/03007995.2014.909394 · Cited by 108 · see citing works

Adeno-associated Virus (AAV) Assembly-Activating Protein Is Not an Essential Requirement for Capsid Assembly of AAV Serotypes 4, 5, and 11

Earley, L. F., Powers, J. M., Adachi, K., Baumgart, J. T., Meyer, N. L., Xie, Q., Chapman, M. S., & Nakai, H. (2017) · Journal of Virology · doi:10.1128/jvi.01980-16 · Cited by 108 · see citing works

Long-term retrospective analysis of gene therapy with Alipogene Tiparvovec and its effect on lipoprotein lipase deficiency-induced pancreatitis

Gaudet, D., Stroes, E. S., Méthot, J., Brisson, D., Tremblay, K., Bernelot Moens, S. J., Iotti, G., Rastelletti, I., Ardigo, D., Corzo, D., et al. (2016) · Human Gene Therapy · doi:10.1089/hum.2015.158 · Cited by 100 · see citing works

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

Salmon, F., Grosios, K., & Petry, H. (2013) · Expert Review of Clinical Pharmacology · doi:10.1586/17512433.2014.852065 · Cited by 93 · see citing works

Lipid Lowering Therapy: An Era Beyond Statins

Abdul‐Rahman, T., Bukhari, S. M. A., Cantu‐Herrera, E., Awuah, W. A., Lawrence, J. A., de Andrade, H., Patel, N., Shah, R. M., Shaikh, R., Capriles, C. A. A., et al. (2022) · Current Problems in Cardiology · doi:10.1016/j.cpcardiol.2022.101342 · Cited by 91 · see citing works

Adeno-associated Virus as a Mammalian DNA Vector

Salganik, M., Hirsch, M. L., & Samulski, R. J. (2015) · Microbiology Spectrum · doi:10.1128/microbiolspec.mdna3-0052-2014 · Cited by 88 · see citing works

Update on Screening, Etiology, and Treatment of Dyslipidemia in Children

Bamba, V. (2014) · The Journal of Clinical Endocrinology & Metabolism · doi:10.1210/jc.2013-3860 · Cited by 80 · see citing works

AAV-ID: A Rapid and Robust Assay for Batch-to-Batch Consistency Evaluation of AAV Preparations

Pacouret, S., Bouzelha, M., Shelke, R., Andres-Mateos, E., Xiao, R., Maurer, A., Mevel, M., Turunen, H., Barungi, T., Penaud-Budloo, M., et al. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.04.001 · Cited by 69 · see citing works

Apolipoprotein B-48

Nakajima, K., Nagamine, T., Fujita, M., Ai, M., Tanaka, A., & Schaefer, E. J. (2014) · Advances in clinical chemistry · doi:10.1016/b978-0-12-800263-6.00003-3 · Cited by 67 · see citing works

Advanced genetic therapies for the treatment of Rett syndrome: state of the art and future perspectives

Palmieri, M., Pozzer, D., & Landsberger, N. (2023) · Frontiers in Neuroscience · doi:10.3389/fnins.2023.1172805 · Cited by 65 · see citing works

Emerging strategies of targeting lipoprotein lipase for metabolic and cardiovascular diseases

Geldenhuys, W. J., Li, L., Darvesh, A. S., & Sadana, P. (2016) · Drug Discovery Today · doi:10.1016/j.drudis.2016.10.007 · Cited by 64 · see citing works

Next-generation muscle-directed gene therapy by in silico vector design

Sarcar, S., Tulalamba, W., Rincón, M. Y., Tipanee, J., Pham, H. Q., Evens, H., Boon, D., Samara-Kuko, E., Keyaerts, M., Loperfido, M., et al. (2019) · Nature Communications · doi:10.1038/s41467-018-08283-7 · Cited by 59 · see citing works

Oligonucleotide conjugated multi-functional adeno-associated viruses

Katrekar, D., Moreno, A. M., Chen, G., Worlikar, A., & Mali, P. (2018) · Scientific Reports · doi:10.1038/s41598-018-21742-x · Cited by 57 · see citing works

Diabetic dyslipidaemia

Soran, H., Schofield, J. D., Adam, S., & Durrington, P. N. (2016) · Current Opinion in Lipidology · doi:10.1097/mol.0000000000000318 · Cited by 57 · see citing works

Novel Chimeric Gene Therapy Vectors Based on Adeno-Associated Virus and Four Different Mammalian Bocaviruses

Fakhiri, J., Schneider, M. A., Puschhof, J., Stanifer, M. L., Schildgen, V., Holderbach, S., Voß, Y., Andari, J. E., Schildgen, O., Boulant, S., et al. (2019) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2019.01.003 · Cited by 57 · see citing works

PPAR-α agonists are still on the rise: an update on clinical and experimental findings

Ferri, N., Corsini, A., Sirtori, C. R., & Ruscica, M. (2017) · Expert Opinion on Investigational Drugs · doi:10.1080/13543784.2017.1312339 · Cited by 56 · see citing works

From Mutation Identification to Therapy: Discovery and Origins of the First Approved Gene Therapy in the Western World

Kastelein, J. J. P., Ross, C. J., & Hayden, M. R. (2013) · Human Gene Therapy · doi:10.1089/hum.2013.063 · Cited by 56 · see citing works

Controlled release strategies for rAAV-mediated gene delivery

Rey‐Rico, A., & Cucchiarini, M. (2015) · Acta Biomaterialia · doi:10.1016/j.actbio.2015.10.015 · Cited by 52 · see citing works

Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction

Muhuri, M., Zhan, W., Maeda, Y., Li, J., Lotun, A., Chen, J., Sylvia, K., Dasgupta, I., Arjomandnejad, M., Nixon, T., et al. (2021) · Frontiers in Immunology · doi:10.3389/fimmu.2021.674242 · Cited by 50 · see citing works

Alipogene tiparvovec: gene therapy for lipoprotein lipase deficiency

Wierzbicki, A. S., & Viljoen, A. (2012) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.2013.738663 · Cited by 48 · see citing works

Centronuclear myopathies under attack: A plethora of therapeutic targets

Tasfaout, H., Cowling, B. S., & Laporte, J. (2018) · Journal of Neuromuscular Diseases · doi:10.3233/jnd-180309 · Cited by 46 · see citing works

Pharmacological treatment options for severe hypertriglyceridemia and familial chylomicronemia syndrome

Chaudhry, R., Viljoen, A., & Wierzbicki, A. S. (2018) · Expert Review of Clinical Pharmacology · doi:10.1080/17512433.2018.1480368 · Cited by 46 · see citing works

Novel therapeutics in hypertriglyceridemia

Gryn, S. E., & Hegele, R. A. (2015) · Current Opinion in Lipidology · doi:10.1097/mol.0000000000000231 · Cited by 44 · see citing works

Pathogenic classification of LPL gene variants reported to be associated with LPL deficiency

Rodrigues, R., Artieda, M., Tejedor, D., Martínez, A., Konstantinova, P. S., Petry, H., Meyer, C., Corzo, D., Sundgreen, C., Klör, H. U., et al. (2015) · Journal of clinical lipidology · doi:10.1016/j.jacl.2015.12.015 · Cited by 44 · see citing works

Suppression of heterotopic ossification in fibrodysplasia ossificans progressiva using AAV gene delivery

Yang, Y.-S., Kim, J., Xie, J., Chaugule, S. R., Lin, C., Ma, H., Hsiao, E. C., Hong, J., Chun, H., Shore, E. M., et al. (2022) · Nature Communications · doi:10.1038/s41467-022-33956-9 · Cited by 43 · see citing works

Extra-adrenal induction of <i>Cyp21a1</i> ameliorates systemic steroid metabolism in a mouse model of congenital adrenal hyperplasia

Naiki, Y., Miyado, M., Horikawa, R., Katsumata, N., Onodera, M., Pang, S., Ogata, T., & Fukami, M. (2016) · Endocrine Journal · doi:10.1507/endocrj.ej16-0112 · Cited by 40 · see citing works

Molecular analysis of chylomicronemia in a clinical laboratory setting: Diagnosis of 13 cases of lipoprotein lipase deficiency

Martín-Campos, J. M., Julve, J., Roig, R., Martínez, S., Errico, T. L., Martínez-Couselo, S., Escolá‐Gil, J. C., Méndez‐González, J., & Blanco‐Vaca, F. (2013) · Clinica Chimica Acta · doi:10.1016/j.cca.2013.11.025 · Cited by 37 · see citing works

Hypertriglyceridemia-Related Pancreatitis In Patients With Type 2 Diabetes: Links And Risks

Shemesh, E., & Zafrir, B. (2019) · Diabetes Metabolic Syndrome and Obesity · doi:10.2147/dmso.s188856 · Cited by 38 · see citing works

Adeno-associated viral vector integration: implications for long-term efficacy and safety

Batty, P. A., & Lillicrap, D. (2024) · Journal of Thrombosis and Haemostasis · doi:10.1016/j.jtha.2024.07.012 · Cited by 36 · see citing works

Concise Review: The Potential Use of Intestinal Stem Cells to Treat Patients with Intestinal Failure

Hong, S. N., Dunn, J., Stelzner, M. G., & Martı́n, M. G. (2016) · Stem Cells Translational Medicine · doi:10.5966/sctm.2016-0153 · Cited by 36 · see citing works

Safety and efficacy of therapies for chylomicronemia

Shamsudeen, I., & Hegele, R. A. (2022) · Expert Review of Clinical Pharmacology · doi:10.1080/17512433.2022.2094768 · Cited by 37 · see citing works

Gene Therapy in Lipoprotein Lipase Deficiency: Case Report on the First Patient Treated with Alipogene Tiparvovec Under Daily Practice Conditions

Kassner, U., Hollstein, T., Grenkowitz, T., Wühle-Demuth, M., Salewsky, B., Demuth, I., Dippel, M., & Steinhagen‐Thiessen, E. (2018) · Human Gene Therapy · doi:10.1089/hum.2018.007 · Cited by 35 · see citing works

Manufacturing of recombinant adeno-associated viral vectors: new technologies are welcome

Ayuso, E. (2016) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2015.49 · Cited by 34 · see citing works

High-Throughput Dissection of AAV–Host Interactions: The Fast and the Curious

Herrmann, A.-K., & Grimm, D. (2018) · Journal of Molecular Biology · doi:10.1016/j.jmb.2018.05.022 · Cited by 33 · see citing works

Pharmacological Inhibition of Monoacylglycerol O-Acyltransferase 2 Improves Hyperlipidemia, Obesity, and Diabetes by Change in Intestinal Fat Utilization

Take, K., Mochida, T., Maki, T., Satomi, Y., Hirayama, M., Nakakariya, M., Amano, N., Adachi, R., Sato, K., Kitazaki, T., et al. (2016) · PLoS ONE · doi:10.1371/journal.pone.0150976 · Cited by 34 · see citing works

The SUMOylation Pathway Restricts Gene Transduction by Adeno-Associated Viruses

Hölscher, C., Sonntag, F., Henrich, K., Chen, Q., Beneke, J., Matula, P., Rohr, K., Kaderali, L., Beil, N., Erfle, H., et al. (2015) · PLoS Pathogens · doi:10.1371/journal.ppat.1005281 · Cited by 33 · see citing works

Therapeutic targets of triglyceride metabolism as informed by human genetics

Bauer, R. C., Khetarpal, S. A., Hand, N. J., & Rader, D. J. (2016) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2016.02.005 · Cited by 32 · see citing works

Causes, clinical findings and therapeutic options in chylomicronemia syndrome, a special form of hypertriglyceridemia

Paragh, G., Németh, Á., Harangi, M., Banach, M., & Fülöp, P. (2022) · Lipids in Health and Disease · doi:10.1186/s12944-022-01631-z · Cited by 34 · see citing works

The burden of familial chylomicronemia syndrome in Canadian patients

Gaudet, D., Stevenson, M. T., Komari, N., Trentin, G. A., Crowson, C., Hadker, N., & Bernard, S. (2020) · Lipids in Health and Disease · doi:10.1186/s12944-020-01302-x · Cited by 29 · see citing works

Quantile-dependent expressivity of postprandial lipemia

Williams, P. T. (2020) · PLoS ONE · doi:10.1371/journal.pone.0229495 · Cited by 28 · see citing works

Lipoprotein abnormalities in compound heterozygous lipoprotein lipase deficiency after treatment with a low-fat diet and orlistat

Blackett, P. R., Tryggestad, J. B., Krishnan, S., Li, S., Xu, W., Alaupovic, P., Quiroga, C., & Copeland, K. C. (2012) · Journal of clinical lipidology · doi:10.1016/j.jacl.2012.11.006 · Cited by 27 · see citing works

Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases

Lin, C., Greenblatt, M. B., Gao, G., & Shim, J. (2024) · Human Gene Therapy · doi:10.1089/hum.2024.022 · Cited by 25 · see citing works

Current AAV-mediated gene therapy in sensorineural hearing loss

Qi, J., Fu, X., Zhang, L., Tan, F., Li, N., Sun, Q., Hu, X., He, Z., Xia, M., & Chai, R. (2022) · Fundamental Research · doi:10.1016/j.fmre.2022.08.015 · Cited by 24 · see citing works

Efficacy and safety of lomitapide in familial chylomicronaemia syndrome

Cefalu, A. B., D’Erasmo, L., Iannuzzo, G., Noto, D., Giammanco, A., Montali, A., Zambon, A., Forte, F., Suppressa, P., Giannini, S., et al. (2022) · Atherosclerosis · doi:10.1016/j.atherosclerosis.2022.08.017 · Cited by 24 · see citing works

Treatment of primary hypertriglyceridemia states – General approach and the role of extracorporeal methods

Stefanutti, C., & Julius, U. (2015) · Atherosclerosis Supplements · doi:10.1016/j.atherosclerosissup.2015.02.017 · Cited by 23 · see citing works

The role of registries in rare genetic lipid disorders: Review and introduction of the first global registry in lipoprotein lipase deficiency

Steinhagen‐Thiessen, E., Stroes, E. S. G., Soran, H., Johnson, C., Moulin, P., Iotti, G., Zibellini, M., Ossenkoppele, B., Dippel, M., & Averna, M. R. (2016) · Atherosclerosis · doi:10.1016/j.atherosclerosis.2016.08.023 · Cited by 23 · see citing works

New trends in articular cartilage repair

Cucchiarini, M., Henrionnet, C., Mainard, D., Pinzano, A., & Madry, H. (2015) · Journal of Experimental Orthopaedics · doi:10.1186/s40634-015-0026-0 · Cited by 21 · see citing works

Combining Engineered Nucleases with Adeno-associated Viral Vectors for Therapeutic Gene Editing

Epstein, B. E., & Schaffer, D. V. (2017) · Advances in experimental medicine and biology · doi:10.1007/978-3-319-63904-8_2 · Cited by 20 · see citing works

Dietary fatty acid metabolism in prediabetes

Noll, C., & Carpentier, A. C. (2016) · Current Opinion in Lipidology · doi:10.1097/mol.0000000000000369 · Cited by 20 · see citing works

Directed Evolution of AAV Targeting Primate Retina by Intravitreal Injection Identifies R100, a Variant Demonstrating Robust Gene Delivery and Therapeutic Efficacy in Non-Human Primates

Kotterman, M., Beliakoff, G., Croze, R., Vazin, T., Schmitt, C., Szymanski, P., Leong, M., Quezada, M., Holt, J., Barglow, K., et al. (2021) · doi:10.1101/2021.06.24.449775 · Cited by 20 · see citing works

AAV-Mediated Targeting of the Activin A-ACVR1R206H Signaling in Fibrodysplasia Ossificans Progressiva

Yang, Y.-S., Lin, C., Mā, H., Xie, J., Kaplan, F. S., Gao, G., & Shim, J. (2023) · Biomolecules · doi:10.3390/biom13091364 · Cited by 21 · see citing works

Gene-based therapies in lipidology

Gaudet, D., & Brisson, D. (2015) · Current Opinion in Lipidology · doi:10.1097/mol.0000000000000240 · Cited by 19 · see citing works

Chitosan-Based Systems for Gene Delivery

Sharma, D., Arora, S., Rodrigues, B. D. S., Lakkadwala, S., Banerjee, A., & Singh, J. (2019) · Springer eBooks · doi:10.1007/978-981-15-0263-7_8 · Cited by 18 · see citing works

Tracers and Imaging of Fatty Acid and Energy Metabolism of Human Adipose Tissues

Carpentier, A. C. (2023) · Physiology · doi:10.1152/physiol.00012.2023 · Cited by 18 · see citing works

Viral Vectors in Gene Replacement Therapy

Minskaia, E., Galieva, A., Egorov, A. D., Ivanov, R. A., & Karabelsky, A. V. (2023) · Biochemistry (Moscow) · doi:10.1134/s0006297923120179 · Cited by 18 · see citing works

Novel therapies for severe dyslipidemia originating from human genetics

Gaudet, D. (2016) · Current Opinion in Lipidology · doi:10.1097/mol.0000000000000281 · Cited by 18 · see citing works

Triglyceride Lowering Drugs

Feingold, K. R. (2017) · Europe PMC (PubMed Central) · Cited by 18 · see citing works

Progress toward improved therapies for inborn errors of metabolism

Ginocchio, V. M., & Brunetti‐Pierri, N. (2015) · Human Molecular Genetics · doi:10.1093/hmg/ddv418 · Cited by 18 · see citing works

Regulatory Oversight of Cell and Gene Therapy Products in Canada

Ridgway, A. A. G., Agbanyo, F. R., Wang, J., & Rosu‐Myles, M. (2015) · Advances in experimental medicine and biology · doi:10.1007/978-3-319-18618-4_3 · Cited by 17 · see citing works

An overview of persistent chylomicronemia: much more than meets the eye

Larouche, M., Watts, G. F., Ballantyne, C. M., & Gaudet, D. (2025) · Current Opinion in Endocrinology Diabetes and Obesity · doi:10.1097/med.0000000000000903 · Cited by 17 · see citing works

AAV-Mediated ApoC2 Gene Therapy: Reversal of Severe Hypertriglyceridemia and Rescue of Neonatal Death in ApoC2-Deficient Hamsters

Yang, C., Tian, W., Ma, S., Guo, M., Lin, X., Gao, F., Dong, X., Gao, M., Wang, Y., Liu, C., et al. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.07.011 · Cited by 15 · see citing works

Preclinical Development and Characterization of Novel Adeno-Associated Viral Vectors for the Treatment of Lipoprotein Lipase Deficiency

Mehta, N., Gilbert, R., Chahal, P. S., Moreno, M., Nassoury, N., Coulombe, N., Lytvyn, V., Mercier, M., Fatehi, D., Lin, W., et al. (2023) · Human Gene Therapy · doi:10.1089/hum.2023.075 · Cited by 15 · see citing works

Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles

Eekhoff, E. M. W., de Ruiter, R. D., Smilde, B. J., Schoenmaker, T., de Vries, T. J., Netelenbos, J. C., Hsiao, E. C., Scott, C., Haga, N., Grunwald, Z., et al. (2022) · Human Gene Therapy · doi:10.1089/hum.2022.023 · Cited by 15 · see citing works

Angiotensin 1-7 Overexpression Mediated by a Capsid-optimized AAV8 Vector Leads to Significant Growth Inhibition of Hepatocellular Carcinoma In vivo

Mao, Y., Pei, N., Chen, X., Chen, H., Yan, R., Bai, N., Li, A. I., Li, J., Zhang, Y., Du, H., et al. (2018) · International Journal of Biological Sciences · doi:10.7150/ijbs.22235 · Cited by 14 · see citing works

Recent developments in pharmacotherapy for hypertriglyceridemia: what’s the current state of the art?

Florentin, M., Kostapanos, M. S., Anagnostis, P. G., & Liamis, G. L. (2019) · Expert Opinion on Pharmacotherapy · doi:10.1080/14656566.2019.1691523 · Cited by 14 · see citing works

A Novel Method for the Quantification of Adeno-Associated Virus Vectors for RNA Interference Applications Using Quantitative Polymerase Chain Reaction and Purified Genomic Adeno-Associated Virus DNA as a Standard

Wagner, A., Röhrs, V., Kedzierski, R., Fechner, H., & Kurreck, J. (2013) · Human Gene Therapy Methods · doi:10.1089/hgtb.2013.095 · Cited by 13 · see citing works

Gene therapy for lipoprotein lipase deficiency

Gaudet, D., Méthot, J., & Kastelein, J. (2012) · Current Opinion in Lipidology · doi:10.1097/mol.0b013e3283555a7e · Cited by 178 · see citing works

Abstract 2093: AAV1-LPLS447X Gene Therapy With Alipogene Tiparvovec Prevents Pancreatitis in Lipoprotein Lipase Deficiency

Franssen, R., Gaudet, D., Stroes, E. S. G., Kastelein, J. J. P., Brisson, D., Rousseau, S., de Wal, J., & van Deventer, S. J. H. (2009) · Circulation · Cited by 1 · see citing works

Alipogene Tiparvovec: A Review of Its Use in Adults with Familial Lipoprotein Lipase Deficiency

Scott, L. J. (2015) · Drugs · doi:10.1007/s40265-014-0339-9 · Cited by 130 · see citing works

Alipogene tiparvovec for the treatment of lipoprotein lipase deficiency

Haddley, K. (2013) · Drugs of today · doi:10.1358/dot.2013.49.3.1937398 · Cited by 22 · see citing works

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

Gaudet, D., Méthot, J., Déry, S., Brisson, D., Essiembre, C., Tremblay, G., Tremblay, K., de Wal, J., Twisk, J., van den Bulk, N., et al. (2013) · Gene Therapy · doi:10.1038/gt.2012.43 · Cited by 404 · see citing works