AAV Technology: Delivery Vehicle of Choice for Gene Therapy

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Adeno-associated viruses (AAVs) are attractive delivery vectors in the field of gene therapy. A team from the National Institutes of Health (NIH) developed AAV5-based vectors for delivering gene therapy products into parts of human bodies. Gene therapy is on the brink of becoming a common medical practice; however, developing safe and effective gene therapy products has been challenging. One major issue has been finding a delivery vector to target the diseased tissues in the body without devastating side effects.

Glybera®: First Gene Therapy Recommended for Approval in the West

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On July 20, 2012, a committee of the European Medicines Agency recommended for regulatory approval the first gene therapy using an adeno-associated virus vector to treat lipoprotein lipase deficiency (LPLD), a very rare genetic disease. With a normal diet, patients lacking sufficient levels of lipoprotein lipase have abnormally high serum triglycerides and high levels of very low-density lipoprotein (VLDL), resulting in acute severe pancreatitis and chronic conditions associated with high levels of VLDL, such as cardiovascular diseases. 

Interleukin-2 Receptor Gamma Deficient Mice, Widely Used Research Tools

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In 1995, Dr. Warren J. Leonard of the National Heart, Lung, and Blood Institute (NHLBI), National Institutes of Health (NIH), reported the creation of an interleukin-2 receptor gamma chain (IL-2Rg) deficient mouse. His lab also demonstrated earlier that mutations in the gene encoding IL-2Rg result in X-linked severe combined immunodeficiency (XSCID) and that IL-2Rg is a subunit common to multiple cytokine receptors. The IL-2Rgdeficient mice created by Dr. Leonard provide a unique animal model, the first of its kind, to study XSCID and other immune deficiencies.

NIH International Technology Transfer Mentoring Program

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Medical research to combat disease is now a global effort and one that now requires an international technology transfer effort to see that new discoveries from research are effectively developed and commercialized to reach patients. While the United States in general, and the National Institutes of Health (NIH) in particular, have long led the way in effective biomedical technology transfer, such is not the case with new or younger transfer programs at research institutions outside the US, especially those in emerging and middle-income countries.

Cristina Thalhammer-Reyero

Dr. Thalhammer-Reyero joined the OTT in 2001. Prior to joining NIH, she was the founder and president of a start-up company, where she developed and patented inventions in the area of computational biology. In earlier years, Dr. Thalhammer-Reyero held various faculty and academic research positions in the areas of immunology, cell biology, cancer biology and biochemistry at Northeastern University, the University of Heidelberg in Germany, Yale University and the University of Vienna in Austria. Dr.

Michael Shmilovich

Michael joined the OTT in 2003. Prior to joining NIH OTT, Michael practiced law at a Washington, DC law firm’s patent division. Before attending law school, he was a biologist at NINDS and NIMH working in the area of clinical genetics. Michael received a degree in law (J.D.) from American University, Washington College of Law, a B.S. in molecular biology and genetics from the University of Michigan, Ann Arbor, and is continuing his scientific education with graduate level immunology courses at the Foundation for the Advanced Education in the Sciences.

Uri Reichman

Dr. Reichman joined the OTT in December 1999. Prior to joining the NIH, Dr. Reichman worked for twenty years (1980-1999) in the biotechnology industry, specializing in the field of medical diagnostics. From 1973 to 1980, Dr. Reichman conducted academic research at Sloan Kettering Institute for Cancer Research in New York. Dr. Reichman is an author of 26 scientific papers and an inventor on several patents. Dr. Reichman received his Ph.D. in Medicinal Chemistry from the Hebrew University in Jerusalem, Israel, and obtained an M.B.A. degree from Iona College in New York.

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