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. Bakshi, S, Diep, T, Willis, BJ, Reyes, R, Wu, GF, Makris, G et al.. Asn674Ile mutation in carbamoyl phosphate synthetase 1 promotes enzyme alteration and hyperammonemia in mice. Dis Model Mech. 2025; :. doi: 10.1242/dmm.052303. PubMed PMID:40421838 .
2. Diep, T, Zhou, W, Reyes, RE, Nitzahn, M, Day, IL, Makris, G et al.. Use of an oversized AAV8 vector for CPS1 deficiency results in long-term survival and ammonia control. Mol Ther Nucleic Acids. 2025;36 (1):102470. doi: 10.1016/j.omtn.2025.102470. PubMed PMID:40083646 PubMed Central PMC11905892.
3. Day, I, Tamboline, M, Lueptow, L, Zhuravka, I, Diep, T, Tkachyova, I et al.. [¹⁸F]FDG-PET and [¹⁸F]MPPF-PET are brain biomarkers for the creatine transporter Slc6a8 loss of function mutation. Sci Rep. 2025;15 (1):7280. doi: 10.1038/s41598-025-92022-8. PubMed PMID:40025148 PubMed Central PMC11873236.
4. Day, IL, Tamboline, M, Lipshutz, GS, Xu, S. Recent developments in translational imaging of in vivo gene therapy outcomes. Mol Ther. 2024; :. doi: 10.1016/j.ymthe.2024.12.049. PubMed PMID:39741403 .
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. medRxiv. 2024; :. doi: 10.1101/2024.09.06.24313213. PubMed PMID:39371127 PubMed Central PMC11451665.

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