Jerry Harb
Biography
Jerry Harb, M.S., is an Associate Specialist at the University of California, Irvine, Department of Pediatrics and a Staff Scientist I at CHOC Children's Hospital Research Institute. His research focuses on genome engineering and rare disease therapeutics, with particular emphasis on lysosomal storage disorders including Pompe disease, Salla disease, and mucopolysaccharidoses. Harb has applied CRISPR-based editing technologies, including homology-directed repair and base editing, to correct pathogenic variants in pediatric metabolic disorders, demonstrating restoration of molecular function in cell-based models.
Harb has executed preclinical studies in murine models of metabolic disorders, conducting longitudinal phenotyping, therapeutic administration, and completing over 1,000 hours of necropsy and tissue processing. He has served as primary scientific liaison to the Institutional Animal Care and Use Committee, ensuring regulatory compliance for research protocols. His work has contributed to the development of novel disease models and the identification of biomarkers for disease progression in lysosomal storage disorders.
Harb has authored peer-reviewed publications in molecular therapy and oncology, including work on base editing correction of the Salla disease SLC17A5 variant and reviews of Wnt signaling pathway inhibitors for cancer therapeutics. He has presented research findings at national conferences and co-organized the Southern California CIRM research retreat. Harb received his M.S. in Biomedical Sciences from Western University of Health Sciences, where his graduate research examined metastasis mechanisms in triple-negative breast cancer using zebrafish xenograft models.
Return to topEducation
- B.S. in Biological Sciences, University of California, Irvine, 2015
- M.S. in Biomedical Sciences, Western University of Health Sciences, 2019
Areas of Expertise
- Translational Rare Disease Therapeutics
- CRISPR Genome Editing
- Lysosomal Storage Disorders
- Murine Phenotyping Models
- Base Editing Correction
- Pompe Disease Biomarkers
- Wnt Signaling Pathway Inhibition
Recent Publications
- Harb JF, Christensen CL, Kan SH, Rha AK, Andrade-Heckman P, Pollard L, Steet R, Huang JY, Wang RY, “Base editing corrects the common Salla disease SLC17A5 c.115C>T variant” (opens in new tab), Molecular Therapy Nucleic Acids, vol. 34, pp. 102022, 2023.
- Chloe L. Christensen, Jerry F. Harb, Allisandra K. Rha, Shih-Hsin Kan, Alexander E. Stover, Raymond Y. Wang, “Generation of an induced pluripotent stem cell line from a patient with free sialic acid storage disorder”, Molecular Genetics and Metabolism, 2022.
- Kan SH, Huang JY, Harb J, Rha A, Dalton ND, Christensen C, Chan Y, Davis-Turak J, Neumann J, Wang RY, “CRISPR-mediated generation and characterization of a Gaa homozygous c.1935C>A (p.D645E) Pompe disease knock-in mouse model recapitulating human infantile onset-Pompe disease” (opens in new tab), Scientific Reports, vol. 12, no. 1, pp. 21576, 2022.
- Allisandra K. Rha, Jeffrey Y. Huang, Shih-hsin Kan, Jerry Harb, Chloe Christensen, Raymond Y. Wang, “Prime editing corrects the c.1826dupA mutation in infantile-onset Pompe disease”, Molecular Genetics and Metabolism, 2022.
- Harb J, Lin PJ, Hao J, “Recent Development of Wnt Signaling Pathway Inhibitors for Cancer Therapeutics” (opens in new tab), Curr Oncol Rep, vol. 21, no. 2, pp. 12, 2019.
Contact Information
Email: jharb@uci.edu
Phone: (949) 300-4522
Address: 1201 W. La Veta, Orange, CA 92868
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Last updated on 5/19/2026.