CHLA scientists receive federal funding to continue their leading-edge work in regenerative research and cell therapeutics

Laura Perin, PhD, and Stefano Da Sacco, PhD, of The Saban Research Institute of Children’s Hospital Los Angeles, investigate how diseases affect kidney cells at a molecular level. The National Institute of Diabetes and Digestive and Kidney Diseases have awarded Dr. Perin and Dr. Da Sacco more than $4 million to study cellular damage and repair in both chronic kidney disease and Alport syndrome, conditions that adversely affect the ability of the body to properly filter blood.

Dr. Da Sacco’s funding will use the team’s breakthrough model called the “glomerulus on a chip” to study the mechanisms of autoimmune diseases of primary membranous nephropathy. Diseases like these damage the cells that make up the kidney’s filtration barrier. Dr. Da Sacco’s studies are aimed at determining which molecules inside these cells are activated during injury. This is a critical step needed for the eventual development of medications and treatments for diseases such as primary membranous neuropathy.

Dr. Perin will study the therapeutic potential of human extracellular vesicles (EVs). EVs are small particles that cells release to communicate with one another and repair damage in nearby cells. Her experiments show that EVs can offer protection in a preclinical model for Alport syndrome, which causes irreversible damage to the cells that make up the kidney’s filtration barrier.

These studies are part of a larger research approach that focuses on regenerative medicine at Children’s Hospital Los Angeles. Dr. Perin and Dr. Da Sacco work in CHLA’s GOFARR Laboratory for Organ Regenerative Research and Cell Therapeutics in Urology, which focuses on tissue engineering, cell therapies and discovering treatments for kidney disease. Their work is supported by multiple core facilities at CHLA, including the Extracellular Vesicle Core, which offers expertise and leading-edge technologies to support EV research.

Source – Children’s Hospital Los Angeles

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