Research Highlights

Our research focuses on the development of gene and cell-based therapies for the treatment of metabolic diseases of the liver that commonly result in the development of intellectual disabilities in children. We have two active research directions:

  1. Development of gene-based (adeno-associated virus, mRNA) therapies to restore nitrogen metabolism and ureagenesis.
  2. Exploring the underlying neurobiology in arginase deficiency to gain a greater understanding of the neuropathology that exists in this enzyme deficiency.

Featured Publications

Lee PC, Truong B, Vega-Crespo A, Gilmore WB, Hermann K, Angarita SA, Tang JK, Chang KM, Wininger AE, Lam AK, Schoenberg BE, Cederbaum SD, Pyle AD, Byrne JA, Lipshutz GS. Restoring Ureagenesis in Hepatocytes by CRISPR/Cas9-mediated Genomic Addition to Arginase-deficient Induced Pluripotent Stem Cells. Mol Ther Nucleic Acids. 2016 Nov 29;5(11).

Cantero G, Liu XB, Mervis RF, Lazaro MT, Cederbaum SD, Golshani P, Lipshutz GS. Rescue of the Functional Alterations of Motor Cortical Circuits in Arginase Deficiency by Neonatal Gene Therapy. J Neurosci. 2016 Jun 22;36(25):6680-90.

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