Felix Nitschke, Ph.D. Titles and Appointments Assistant Professor Schools Medical School | Graduate School Departments Pediatrics | Biochemistry Graduate Programs Biological Chemistry, Neuroscience Biography Dr. Nitschke received his Ph.D. from the University of Potsdam, Germany, in 2013. He performed his doctoral work in the lab of Dr. Martin Steup, an internationally accomplished expert in the field of starch metabolism. This research area is of great relevance to an area of medicine, namely that of glycogen storage disorders. Dr. Nitschke focused his work on the investigation of Lafora disease (LD), where instead of synthesizing exclusively soluble glycogen, insoluble, starch-like glycogen particles accumulate and drive the severe form of progressive childhood-onset epilepsy. He completed his post-doc in the gene therapy lab of Dr. Berge Minassian at The Hospital for Sick Children Research Institute, Toronto, Canada, and is now Assistant Professor at UT Southwestern Medical Center in the Departments of Pediatrics and Biochemistry. Dr. Nitschke is an expert in carbohydrate research, combining his interest in therapeutic application with a deep understanding of biochemical methodology. He has published several papers in high-ranking peer-reviewed journals and is a frequent speaker at the International Lafora Disease Workshop. In an effort to encourage scientific collaboration and to generally boost understanding of polyglucan metabolism, he published a text book that for the first time integrates enzymology of plant starch and glycogen. His research led to a paradigm change in the field of glycogen metabolism in regards to Lafora disease pathogenesis. He demonstrated that glycogen hyperphosphorylation is a consequence but not the cause of the disease and that it is the abnormal glycogen structure that is critical for the disease. Furthermore, Dr. Nitschke contributed to the development and testing of a therapeutic approach that is based on a fusion of a starch degrading enzyme and diphtheria toxin (DT), a cell-penetrating protein. He could demonstrate that amylase, fused to a non-toxic form of DT, translocates into the cytosol of human cells, is functional and degrades cytosolic glycogen. Cell-penetrating therapeutic proteins provide a promising platform for the treatment of LD and other diseases where abnormal, starch-like glycogen accumulates. Dr. Nitschke’s present work is based on two pillars: 1) basic research for a better understanding of glycogen metabolism, especially regarding the mechanisms that ensure glycogen solubility, and 2) establishment of a novel enzyme replacement platform against multiple rare diseases that aims to overcome the caveats of purely viral delivery approaches. Research Interest Cell Metabolism Enzyme Replacement Glycogen Metabolism (regulation, quality control, related diseases) Glycogen Storage Disorders Novel Gene Therapy Approaches Publications Lafora disease gene therapy: EPM2A but not EPM2B overexpression results in Lafora body formation Alao EO, Sheibani M, Wu J, Marriam U, Evans D, Kasiri S, Verma M, Nitschke S, Nitschke F, Gray SJ, Mitra S, Minassian BA Neurotherapeutics 2026 Mar 23 Neurofilament Light Chain as a Biomarker of Disease Progression in Lafora Disease Muccioli L, Ganceviciute B, Becker F, Minardi R, Tappatà M, Bachhuber F, Alkhatib M, Cirak S, Weishaupt J, Verma M, Tumani H, Wagner J, Messahel S, Nitschke F, Minassian BA, Bisulli F, Brenner D Neurology: Genetics 2025 Dec 11 e200319 Covalently linked phosphate monoesters on alpha-polyglucans reduce substrate affinity of branching enzymes Butler V, Shaaban H, Nasanovsky L, White JK, Hebb O, Jones L, Whiting M, Mukherjee-Roy N, Montalbano AP, Machado Vides CE, Nitschke F, Tetlow IJ Carbohydrate Polymers 2025 Jul 359 Glycogen synthase GYS1 overactivation contributes to glycogen insolubility and malto-oligoglucan-associated neurodegenerative disease Nitschke S, Montalbano AP, Whiting ME, Smith BH, Mukherjee-Roy N, Marchioni CR, Sullivan MA, Zhao X, Wang P, Mount H, Verma M, Minassian BA, Nitschke F EMBO Journal 2025 Mar 44 1379-1413 Neurological, metabolic and inflammatory phenotypes in a mouse model of ECHS1 deficiency Eller MM, Zuberi AR, Fu X, Montalbano AP, Nitschke F, Burgess SC, Lutz C, Bailey RM Brain Communications 2025 7 GDE5/Gpcpd1 activity determines phosphatidylcholine composition in skeletal muscle and regulates contractile force in mice Aisyah R, Ohshima N, Watanabe D, Nakagawa Y, Sakuma T, Nitschke F, Nakamura M, Sato K, Nakahata K, Yokoyama C, Marchioni CR, Kumrungsee T, Shimizu T, Sotomaru Y, Takeo T, Nakagata N, Izumi T, Miura S, Minassian BA, Yamamoto T, Wada M, Yanaka N Communications Biology 2024 Dec 7 Myofiber-type-dependent ‘boulder’ or ‘multitudinous pebble’ formations across distinct amylopectinoses Mitra S, Chen B, Shelton JM, Nitschke S, Wu J, Covington L, Dear M, Lynn T, Verma M, Nitschke F, Fuseya Y, Iwai K, Evers BM, Minassian BA Acta Neuropathologica 2024 Jun 147 Novel Cross-Correction–Enabled Gene Therapy for CDKL5-Deficiency Disorder Nitschke F, Montalbano AP Neurotherapeutics 2022 Oct 19 1878-1882 Glycogen synthase downregulation rescues the amylopectinosis of murine RBCK1 deficiency Nitschke S, Sullivan MA, Mitra S, Marchioni CR, Lee JP, Smith BH, Ahonen S, Wu J, Chown EE, Wang P, Petković S, Zhao X, Digiovanni LF, Perri AM, Israelian L, Grossman TR, Kordasiewicz H, Vilaplana F, Iwai K, Nitschke F, Minassian BA Brain 2022 Jul 145 2361-2377 CRISPR–Cas9-mediated editing of starch branching enzymes results in altered starch structure in Brassica napus Wang L, Wang Y, Makhmoudova A, Nitschke F, Tetlow IJ, Emes MJ Plant Physiology 2022 Apr 188 1866-1886 Results 1-10 of 26 1 2 3 Next Last Books Featured Books Enzymology of Complex Alpha-Glucans (1st ed.). Nitschke, F. (Ed.) (2021). CRC Press. Professional Associations/Affiliations American Society for Biochemistry and Molecular Biology (2020) American Society of Gene and Cell Therapy (2021) Association for Glycogen Storage Disease (2020)
