Repair and regeneration of the nephron
Kidney function is essential for removing the wastes that result from normal cell function and maintaining water and salt balance in our internal tissues. These actions are carried out by…
Preclinical Model for Labeling, Transplant, and In Vivo Imaging of Differentiated Human Embryonic Stem Cells
The derivation and culture of human embryonic stem cells has provided new possibilities for treatment of a wide variety of human diseases because these cells have the potential to help…
Providing a cure for sphingosine phosphate lyase insufficiency syndrome (SPLIS) through adeno-associated viral mediated SGPL1 gene therapy
Research Objective AAV-SPL 2.0 is a gene therapy cure for SPLIS, a lethal childhood disorder of metabolism that causes kidney failure. Our gene therapy may also work in more common…
Enhanced Branching Morphogenesis and Pluripotent Cell Lineage Differentiation for Pediatric Regenerative Therapies
Research Objective Approximately 20,000 babies are born annually with kidney disease; the long-term outcome is poor. These studies address new ways to develop mini-kidney structures for transplantation to induce repair.…
CIRM ASCEND Center – Advancing Stem Cell Education and Novel Discoveries
The proposed CIRM ASCEND Center provides cutting-edge organoid-based services, offering consultation, optimized protocols, and genomic analysis. We also offer comprehensive training, workshops, and access to data, empowering researchers across California…
Development of in vitro and in vivo functional human synthetic kidney organoid (hSKO) model as a platform technology for kidney research
Research Objective Development of a human stem cell-derived, spatially-patterned, mature and functional human synthetic kidney organoid (hSKO) model as a platform technology for basic and translational kidney research. Impact Lack…
Andrew McMahon, USC – CIRM Stem Cell #SciencePitch
Produced by the University of Southern California