Intellectual and Developmental Disabilities Research Center

Members

Lipshutz, Gerald, M.D.

Research

Gene and Cellular Therapy for Genetic Disorders by In utero and Neonatal Therapy

Appointments

  • Member, Liver Transplantation
  • JCCC Tumor Immunology Program Area
  • Pfleger Liver Institute
  • Assistant Professor In-Residence, Surgery
  • Endocrinology, Diabetes and Hypertension

Biography

Dr. Lipshutz’s research is directed toward the development of strategies for gene and cellular transfer that may permit the cure of many forms of inherited disease. In these studies, the primary goals are to:

  • develop strategies for the treatment of inherited disease before the clinical onset of phenotype at birth
  • to examine if tolerance to foreign transgene products can be induced by delivery and expression of the protein during immunologic development
  • to track hematopoeitic and hepatic cells transplanted before birth and determine their final fate as a method to develop cellular therapy

Publications

  1. Bogoniewski, AA, Gonzalez, MF, Reyes, RE, Rivera, SF, Taylor, C, Schurr, T et al.. Machine Learning-Driven Automated Culture Reveals Similar Performance of mTeSR+ and StemFlex in Human iP11N Pluripotent Stem Cells. Cells. 2026;15 (17):. doi: 10.3390/cells15171533. PubMed PMID:42738827 PubMed Central PMC13565254.
  2. Xu, R, Diep, T, Singh, K, Bakshi, S, Wu, GF, Lu, M et al.. A hybrid piggyBac AAV-transposon and LNP-transposase for treating arginase deficiency: A 100-fold dose reduction compared to AAV alone. Mol Ther. 2026;34 (8):4449-4465. doi: 10.1016/j.ymthe.2026.04.045. PubMed PMID:42050931 PubMed Central PMC13464163.
  3. Meera, P, Uusi-Oukari, M, Wallner, M, Lipshutz, GS. Guanidino compounds with native GABA(A) δ receptor selectivity: a tale of homeostatic compensation in δ-KO mice. BMC Neurosci. 2025;27 (1):3. doi: 10.1186/s12868-025-00987-z. PubMed PMID:41372746 PubMed Central PMC12801676.
  4. Diep, T, Lipshutz, GS. Evaluation of SLC6A8 species conservation and the effect of pathogenic variants on creatine transport. HGG Adv. 2025;6 (4):100489. doi: 10.1016/j.xhgg.2025.100489. PubMed PMID:40781773 PubMed Central PMC12398244.
  5. Nasseri Moghaddam, Z, Reinhardt, EK, Thurm, A, Potter, BK, Smith, M, Graham, C et al.. Establishing a core outcome set for creatine transporter deficiency and guanidinoacetate methyltransferase deficiency. Orphanet J Rare Dis. 2025;20 (1):408. doi: 10.1186/s13023-025-03900-3. PubMed PMID:40775643 PubMed Central PMC12333098.
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