Skip to Main Content
Xin Chen, Ph.D.

Xin Chen, Ph.D.

Titles and Appointments

Assistant Professor

Schools
Medical School
Departments
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
    Focused ultrasound widely broadens AAV-delivered Cas9 distribution and activity
    Gumusgoz E, Kasiri S, Youssef I, Verma M, Chopra R, Villarreal Acha D, Wu J, Marriam U, Alao E, Chen X, Guisso DR, Gray SJ, Shah BR, Minassian BA Gene Therapy 2025 May 32 237-245
    Lumbar Intrathecal Injection of Gene Therapy Vectors for Central Nervous System Targeting in Mice and Rats
    Boitnott A, Rioux M, Chen X, Gray S Journal of Visualized Experiments 2025 May 2025-May
    Direct Vagus Nerve Injection Protocol For Rats
    Regmi NL, Chen X, Bailey RM, Gray SJ Journal of Visualized Experiments 2025 Mar 2025-March
    Riboflavin transporter deficiency: AAV9-SLC52A2 gene therapy as a new therapeutic strategy
    Mei C, Magliocca V, Chen X, Massey K, Gonzalez-Cordero A, Gray SJ, Tartaglia M, Bertini ES, Corti S, Compagnucci C Frontiers in Cellular Neuroscience 2025 19
    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 Nature medicine 2024 Jul 30 1882-1887
    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 Therapy 2024 May 31 234-241
    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 2023 Aug 14
    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 Journal of Clinical Investigation 2023 May 133
    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 Human gene therapy 2023 Feb 34 94-111
    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
  • Books

    Featured 

    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 Award
      ORS Annual Meeting - Washington, DC (2005)
    • ASBMR Young Investigator Award
      American Society for Bone and Mineral Research (2004)
    • Plenary Poster - ASBMR Annual Meeting
      American 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)