J. Andrea Sierra-Delgado, MD, MSc

Physical Medicine and Rehabilitation

Assistant Research Professor

573-882-3101

Profile

J. Andrea Sierra-Delgado, MD, MSc, is a gene therapy researcher focused on investigating rare neurological and neuromuscular disorders to drive therapeutic discovery through the development of innovative in vitro disease models and gene therapy strategies. Her research aims to elucidate genotype–phenotype correlations in neurodegenerative, muscular and seizure/autism disorders, uncovering disease mechanisms to translate findings into clinical applications. Early in her career, Dr. Sierra-Delgado provided the first evidence of natural exposure to adeno-associated virus in Latin America and revealed the influence of socioeconomic factors on neutralizing antibody prevalence — critical considerations for the advancement of gene therapy. Building on this foundation, she led multi-centered projects at Nationwide Children’s Hospital that resulted in FDA approval for a Phase I/IIa clinical trial targeting IGHMBP2-related disorders, highlighting her ability to bridge preclinical research and clinical translation. Dr. Sierra-Delgado’s research focuses on developing advanced in vitro modeling systems to study disease mechanisms at the cellular level, utilizing high-throughput screening approaches to optimize AAV9 gene therapy testing and refine personalized therapeutic strategies. She also brings extensive experience in IND-enabling preclinical studies, including proof-of-concept and toxicology assessments. Currently, as an assistant research professor under the mentorship of Smita Saxena, PhD, at the University of Missouri School of Medicine, Dr. Sierra-Delgado continues to push the boundaries of translational gene therapy research, applying cutting-edge techniques to accelerate the development of treatments for children and families affected by rare neurological and neuromuscular conditions.

Academic Information

Assistant Research Professor

Office

1030 Hitt Street
Columbia, MO 65211
United States

P. 573-882-3101

Research Interests

  •  Genotype–phenotype correlations and disease mechanisms in neurodevelopmental, neurodegenerative, neuromuscular, and seizure/autism spectrum disorders
  • Development of in vitro disease models for studying pathophysiology and therapeutic screening
  • Generation and Optimization of AAV-based gene therapy strategies
  • IND-enabling proof-of-concept and toxicology studies for gene therapy translation
  • Advancement of personalized and targeted treatments for rare neurological and neuromuscular disorders

Areas of Expertise

  • Gene Therapy
  • In vitro modelling systems
  • Translational neuroscience.

Education & Training

Degrees

2018 Masters, Universidad Industrial de Santander
2015 MD, Universidad de Los Andes

Awards & Honors

  • Outstanding Poster Award, 27th American Society of Gene & Cell Therapy Annual Meeting, Baltimore, MD, 2024
  • Underrepresented Population Travel Award, 27th American Society of Gene & Cell Therapy Annual Meeting, Baltimore, MD, 2024
  • Chosen as “Describe Your Neuron Like the Allen Institute” Workshop participant-Allen Institute, Seattle, WA, 2024
  • Poster Presentation Award-Neuroscience Research Institute, 2023 Annual Retreat, The Ohio State University, Columbus, OH, 2023
  • Outstanding Research Associate 2021-Clinical Research. The Research Institute Research Retreat Nationwide Children’s Hospital, Columbus, OH, 2022
  • RDD Poster Scholarship. Frank H. Netter MD School of Medicine, Hayden, CT, 2022
  • Best Talk Muscle Center Trainee Research Day. The Ohio State University, Columbus, OH, 2021
  • Best Poster Award. Conference Committee of International Electronic Conference on Brain Sciences, 2020
  • Abstract of Distinction. The Research Institute Research Retreat-t Nationwide Children’s Hospital, Columbus, OH, 2020
  • Meritorious Thesis Score-Master’s Program, Universidad Industrial de Santander, Colombia, 2018
  • Best Clinical Poster-XV National and IX International Human Genetics Congress, Barranquilla, Columbia, 2018
  • Chosen as one of the 70 students from all Latin America to assist to the XII edition of the Latin American School of Human and Medical Genetics (ELAG), Porto Alegre, Brazil, 2016
  • Third-Place-Best Research Protocol, XXVII Colombian Student Congress of Medical Research, Bucaramanga, Colombia, 2016
  • Top Results, National Level, Medicine Program-ICFES SABER PRO exam, Colombia, 2014
  • Full Academic Merit Scholarship, Universidad de Los Andes, Colombia, 2009-2014
  • Biannual Academic Excellence Scholarship, Faculty of Medicine-Universidad de Los Andes, Colombia, 2009
  • Third Place, XVI Colombian National Chemistry Olympiad, Universidad Nacional de Colombia, Colombia, 2008
  • Top 50 Results-National Level-ICFES SABER 11 Exam, Colombia, 2008

Publications

  • Pilotto, F., Smeele, P. H., Scheidegger,., Diab, R., Schobesberger, M., Sierra-Delgado, J. A., & Saxena, S. (2025). Kaempferol enhances ER-mitochondria coupling and p O rotects motor neurons from mitochondrial dysfunction and ER stress in C9ORF72-ALS. Acta Neuropathologica Communications, 13(1), 21.
  • Dennys C.N, Vermudez, SAD, Deacon R.J.M, Sierra-Delgado J.A, Rich K, Zhang X, Buch A, Weiss K, Moxley Y, Rajpal H, Espinoza F.D, Powers, S, Avila A, Rocco G, Congram P, Niswender C, Meyer K.C. MeCP2 gene therapy ameliorates disease phenotype in mouse model for Pitt Hopkins Syndrome. Neurotherapeutics, e00376.
  • Prusty, A. B., Hirmer, A., Sierra-Delgado, J. A., Huber, H., Guenther, U. P., Schlosser, A,.A Dybkov, O., Urlaub, H., Meyer, K., Jablonka, S. & Fischer, U. RNA Helicase Ighmbp2 Regulates THO Complex to Ensure Cellular mRNA Homeostasis. Cell Reports, 43(2), 2024.
  • Sierra-Delgado, J. A., Sinha-Ray, S., Kaleem, A., Ganjibakhsh, M., Parvate, M., Powers, S.,Zhang ,X, Likhite, SB, & Meyer, K. (2023). In Vitro Modeling as a Tool for Testing Therapeutics for Spinal Muscular Atrophy and IGHMBP2-Related Disorders. Biology, 12(6), 867.
  • Schwartz, M. K., Likhite, S., Vetter, T. A., Baird, M. C., McGovern, V., Sierra Delgado, A, Mendel, T, Burghes A,. & Meyer, K. C. (2023). In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humans. Molecular Therapy-Methods & Clinical Development, 30, 16-29.
  • Sierra-Delgado JA, Likhite S, Bautista, P, Gomez-Ochoa S, Echeverria L, Gui E, Vargas C, Serrano N, Meyer K, Rincon M. Prevalence Of Neutralizing Antibodies against Adeno-associated Virus Serotypes 1,2, and 9 in Non-Injected Latin American Patients with Heart Failure- ANVIAS Study. International journal of molecular sciences, 24(6), 5579.
  • Dennys, C. N., Roussel, F., Rodrigo, R., Zhang, X., Sierra-Delgado, A., Hartlaub, A.,et al. & Meyer, K. (2022). CuATSM effectively ameliorates ALS patient astrocyte‐mediated motor neuron toxicity in human in vitro models of amyotrophic lateral sclerosis. Glia. 2022 Oct 10