Gene therapy for lipoprotein lipase deficiency yields promising results


Credit: Mary Ann Liebert, Inc., publishers

New Rochelle, NY, May 1, 2018–During the first 18 months after treatment with ali-pogene tiparvovec, a gene therapy recently approved in Europe to treat lipoprotein lipase deficiency (LPLD), the first patient to receive the treatment had no abdominal pain or episodes of pancreatitis, following a history of 37 pancreatitis attacks. The patient was able to discontinue plasmaphere and described improved quality of life, as reported in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free for download on the Human Gene Therapy website until June 1, 2018.

The article "Gene Therapy in Lipoprotein Lipase Deficiency (LPLD): Case Report on the First Patient Treated with Alipogene Tiparvovec under Daily Practice Conditions" was coauthored by Ursula Kassner, Tim Hollstein, Thomas Grenkowitz, Marion Wühle-Demuth, Bastian Salewsky, Ilja Demuth, Elisabeth Steinhagen-Thiessen, from Chari-té – Universitätsmedizin Berlin, and Michaela Dippel, MD Medscript, Bad Dürheim, Germany.

LPLD is a rare inherited disorder associated with an increased incidence of inflammation of the pancreas due to elevated triglyceride levels. Gene therapy with alipogene tipar-vovec offers a potential cure for LPLD, which is currently treated with plasmapheresis when maintenance of an ultra-low fat diet, use of fibrates, and other triglyceride-lowering therapies prove inadequate. Alipogene tiparvovec is an adeno-associated virus 1 (AAV1) gene therapy administered via intramuscular injections together with immunosuppression.

Uniqure, the manufacturer of alipogene tiparvovec (marketed as Glybera), has ceased post-marketing studies and withdrawn the product from the market in the European Un-ion due to very limited demand for its use. The withdrawal of Glybera was not related to any risk-benefit concerns.

"While Glybera has not continued in commercial sales, it is very gratifying to gene thera-py translational researchers to see a case report of a safe and effective gene therapy prod-uct in clinical use," says Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Worcester, MA.


About the Journal

Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies, is an authoritative peer-reviewed journal published monthly in print and online. Led by Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Human Gene Therapy presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Its companion journals, Human Gene Therapy Methods, published bimonthly, focuses on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, published quarterly, features data relevant to the regulatory review and commercial development of cell and gene therapy products. Tables of contents for all three publications and a free sample issue may be viewed on the Human Gene Therapy website.

About the Publisher

Mary Ann Liebert, Inc., publishers ( is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Nucleic Acid Therapeutics, Tissue Engineering, Stem Cells and Development, and Cellular Reprogramming. Its biotechnology trade magazine, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 80 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.

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