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
- 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.
- Bricker-Anthony, R, Koeberl, DD, Lipshutz, GS, Perna, F. Staying on target in gene and cell therapy. Mol Ther. 2024;32 (9):2801-2802. doi: 10.1016/j.ymthe.2024.08.010. PubMed PMID:39178850 PubMed Central PMC11403208.
- Lum, EL, Pirzadeh, A, Datta, N, Lipshutz, GS, McGonigle, AM, Hamiduzzaman, A et al.. A2/A2B Deceased Donor Kidney Transplantation Using A2 Titers Improves Access to Kidney Transplantation: A Single-Center Study. Kidney Med. 2024;6 (7):100843. doi: 10.1016/j.xkme.2024.100843. PubMed PMID:38947773 PubMed Central PMC11214338.
- Koeberl, D, Schulze, A, Sondheimer, N, Lipshutz, GS, Geberhiwot, T, Li, L et al.. Publisher Correction: Interim analyses of a first-in-human phase 1/2 mRNA trial for propionic acidaemia. Nature. 2024;630 (8017):E13. doi: 10.1038/s41586-024-07646-z. PubMed PMID:38844679 PubMed Central PMC11186770.
- Koeberl, D, Schulze, A, Sondheimer, N, Lipshutz, GS, Geberhiwot, T, Li, L et al.. Author Correction: Interim analyses of a first-in-human phase 1/2 mRNA trial for propionic acidaemia. Nature. 2024;629 (8011):E10. doi: 10.1038/s41586-024-07413-0. PubMed PMID:38649490 .