Stephanie Cherqui
Institution Name:
University of California, San Diego
Total Awards:
2
Award Value:
$17,273,133
Awards as PI
| Program Type | Institution | Grant Title | Award Value |
|---|---|---|---|
| Late Stage Preclinical Projects | University of California, San Diego | Ex vivo transduced autologous human CD34+ hematopoietic stem cells for treatment of cystinosis | $5,273,189 |
| Clinical Trial Stage Projects | University of California, San Diego | Phase 1/2 study for autologous human CD34+ hematopoietic stem cells ex vivo transduced with pCCL-CTNS lentiviral vector for treatment of Cystinosis. | $11,999,944 |
Total: $17,273,133.00 |
Publications
- Front Mol Neurosci 2017 Tunneling Nanotubes and Gap Junctions-Their Role in Long-Range Intercellular Communication during Development, Health, and Disease Conditions. (PubMed: 29089870)
- Sci Transl Med 2017 Transplantation of wild-type mouse hematopoietic stem and progenitor cells ameliorates deficits in a mouse model of Friedreich's ataxia. (PubMed: 29070698)
- Pediatr Nephrol 2018 Potential use of stem cells as a therapy for cystinosis. (PubMed: 29789935)
- PLoS One 2021 Non-invasive intradermal imaging of cystine crystals in cystinosis. (PubMed: 33661986)
- Mol Ther Methods Clin Dev 2020 CRISPR-Cas9 Gene Editing of Hematopoietic Stem Cells from Patients with Friedreich's Ataxia. (PubMed: 32462051)
- Sci Rep 2019 Macrophage polarization impacts tunneling nanotube formation and intercellular organelle trafficking. (PubMed: 31601865)
- Kidney Int 2019 Interaction between galectin-3 and cystinosin uncovers a pathogenic role of inflammation in kidney involvement of cystinosis. (PubMed: 30928021)
- Autophagy 2022 DYNC1LI2 regulates localization of the chaperone-mediated autophagy receptor LAMP2A and improves cellular homeostasis in cystinosis. (PubMed: 34643468)
- Cells 2021 Hematopoietic Stem Cell Gene Therapy for Cystinosis: From Bench-to-Bedside. (PubMed: 34943781)
- Mol Genet Metab 2021 Deficiency of the sedoheptulose kinase (Shpk) does not alter the ability of hematopoietic stem cells to rescue cystinosis in the mouse model. (PubMed: 34823997)