Intellectual and Developmental Disabilities Research Center


Cederbaum, Stephen


Human arginases, mental retardation and gene therapy


  • Director, Medical Genetics Clinic
  • Metabolic Clinic
  • Professor, Pediatrics
  • Chief Emeritus, Pediatric Genetics
  • Professor, Human Genetics


Dr. Andrews leads efforts in basic and translational research on anxiety and depression, and at the nexus of nanoscience and neuroscience.

Andrews’ interdisciplinary research team of neuroscientists, biologists, chemists, and engineers focuses on understanding how the serotonin system and particularly, the serotonin transporter, modulate neurotransmission to influence complex behaviors including anxiety, mood, stress responsiveness, and learning and memory.

Nanomaterials are designed for fundamental studies on neurotransmitter recognition by native and nonnative binding partners (aptamers) and for the development of in vivo nanobiosensors and functionally directed proteomics.


  1. Cederbaum, SD, Edwards, J, Kellmeyer, T, Peters, Y, Steiner, RD. Taste-masked formulation of sodium phenylbutyrate (ACER-001) for the treatment of urea cycle disorders. Mol Genet Metab. 2023;138 (4):107558. doi: 10.1016/j.ymgme.2023.107558. PubMed PMID:37004302 .
  2. Diaz, GA, Bechter, M, Cederbaum, SD. The role and control of arginine levels in arginase 1 deficiency. J Inherit Metab Dis. 2023;46 (1):3-14. doi: 10.1002/jimd.12564. PubMed PMID:36175366 PubMed Central PMC10091968.
  3. Khoja, S, Liu, XB, Truong, B, Nitzahn, M, Lambert, J, Eliav, A et al.. Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency. Mol Ther Nucleic Acids. 2022;28 :859-874. doi: 10.1016/j.omtn.2022.04.012. PubMed PMID:35694211 PubMed Central PMC9156989.
  4. Eno, CC, Barton, SK, Dorrani, N, Cederbaum, SD, Deignan, JL, Grody, WW et al.. Confidential genetic testing and electronic health records: A survey of current practices among Huntington disease testing centers. Mol Genet Genomic Med. 2020;8 (1):e1026. doi: 10.1002/mgg3.1026. PubMed PMID:31701651 PubMed Central PMC6978271.
  5. Truong, B, Allegri, G, Liu, XB, Burke, KE, Zhu, X, Cederbaum, SD et al.. Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency. Proc Natl Acad Sci U S A. 2019;116 (42):21150-21159. doi: 10.1073/pnas.1906182116. PubMed PMID:31501335 PubMed Central PMC6800360.
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