Xin Chen, Ph.D. Assistant Professor School Medical School Department Pediatrics Biography Xin Chen, Ph.D., is an Assistant Professor in the Department of Pediatrics at UT Southwestern Medical Center. He joined the UT Southwestern faculty in 2018 after being in the medical field for more than 20 years. Dr. Chen earned his medical degree at North China Coal Medical College in 1986, followed by a master’s in pathophysiology in 1989 at Hengyang Medical College. In 2000, he completed his Doctor of Philosophy in Biosignal Pathophysiology at Kobe University in Kobe, Japan. He then completed a fellowship at Case Western Reserve University. The recipient of multiple investigator awards for research, Dr. Chen focuses his scientific efforts on developing adeno-associated virus (AAV) based gene therapy treatments for neurological diseases with the goal of translating preclinical results into clinical trials. Research Interest Developing AAV-based gene therapy for neurological diseases Engineering AAV vector to improve efficacy/specificity and reduce immune responses Optimizing manufacturing processes to improve AAV vector quantity and quality Translating preclinical results of gene therapy into clinical trials Publications Featured Publications Intrathecal AAV9/AP4M1 gene therapy for hereditary spastic paraplegia 50 shows safety and efficacy in preclinical studies. Chen X, Dong T, Hu Y, De Pace R, Mattera R, Eberhardt K, Ziegler M, Pirovolakis T, Sahin M, Bonifacino JS, Ebrahimi-Fakhari D, Gray SJ, J Clin Invest 2023 May 133 10 Biodistribution of Adeno-Associated Virus Gene Therapy Following Cerebrospinal Fluid-Directed Administration. Chen X, Lim DA, Lawlor MW, Dimmock D, Vite CH, Lester T, Tavakkoli F, Sadhu C, Prasad S, Gray SJ, Hum Gene Ther 2023 Feb 34 3-4 94-111 AAV9/MFSD8 gene therapy is effective in preclinical models of neuronal ceroid lipofuscinosis type 7 disease. Chen X, Dong T, Hu Y, Shaffo FC, Belur NR, Mazzulli JR, Gray SJ, J Clin Invest 2022 Jan Pre-clinical Gene Therapy with AAV9/AGA in Aspartylglucosaminuria Mice Provides Evidence for Clinical Translation. Chen X, Snanoudj-Verber S, Pollard L, Hu Y, Cathey SS, Tikkanen R, Gray SJ, Mol Ther 2021 03 29 3 989-1000 AAV gene therapy for hereditary spastic paraplegia type 50: a phase 1 trial in a single patient. Dowling JJ, Pirovolakis T, Devakandan K, Stosic A, Pidsadny M, Nigro E, Sahin M, Ebrahimi-Fakhari D, Messahel S, Varadarajan G, Greenberg BM, Chen X, Minassian BA, Cohn R, Bonnemann CG, Gray SJ, Nat Med 2024 Jun CSTB gene replacement improves neuroinflammation, neurodegeneration and ataxia in murine type 1 progressive myoclonus epilepsy. Gumusgoz E, Kasiri S, Verma M, Wu J, Villarreal Acha D, Marriam U, Fyffe-Maricich S, Lin A, Chen X, Gray SJ, Minassian BA, Gene Ther 2023 Dec SMRT Sequencing Enables High-Throughput Identification of Novel AAVs from Capsid Shuffling and Directed Evolution. Casy W, Garza IT, Chen X, Dong T, Hu Y, Kanchwala M, Trygg CB, Shyng C, Xing C, Bunnell BA, Braun SE, Gray SJ, Genes (Basel) 2023 Aug 14 8 Long-term progression of retinal degeneration in a preclinical model of CLN7 Batten disease as a baseline for testing clinical therapeutics. Rowe AA, Chen X, Nettesheim ER, Issioui Y, Dong T, Hu Y, Messahel S, Kayani SN, Gray SJ, Wert KJ, EBioMedicine 2022 Nov 85 104314 Knockout of the CMP-Sialic Acid Transporter SLC35A1 in Human Cell Lines Increases Transduction Efficiency of Adeno-Associated Virus 9: Implications for Gene Therapy Potency Assays. Banning A, Zakrzewicz A, Chen X, Gray SJ, Tikkanen R, Cells 2021 May 10 5 Hemophilia A and B mice, but not VWF-/-mice, display bone defects in congenital development and remodeling after injury. Taves S, Sun J, Livingston EW, Chen X, Amiaud J, Brion R, Hannah WB, Bateman TA, Heymann D, Monahan PE, Sci Rep 2019 10 9 1 14428 Results 1-10 of 27 1 2 3 Next Last Books Featured Books Use of siRNA and antisense knockdown to study regulation of PKA by PKI.. In Handbook of Cell Signaling (Second Edition) Greenfield EM & Chen X (2010). New York, NY, El Sevier Honors & Awards New Investigator Recognition AwardORS Annual Meeting - Washington, DC (2005) ASBMR Young Investigator AwardAmerican Society for Bone and Mineral Research (2004) Plenary Poster - ASBMR Annual MeetingAmerican Society for Bone and Mineral Research (2003) Professional Associations/Affiliations American Society for Bone and Mineral Research (2000-2014) American Society of Gene and Cell Therapy (2014)