News Release 

Children's Hospital Los Angeles investigators awarded $4 million to study kidney diseases

CHLA scientists receive federal funding to continue their leading-edge work in chronic kidney disease and Alport syndrome

Children's Hospital Los Angeles

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.

Dr. Da Sacco's grant provides $1.9 over 5 years (1R01DK123234-01A1) and Dr. Perin was awarded $2.2M over 4 years (1R01DK121037-01A1).

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About Children's Hospital Los Angeles

Founded in 1901, Children's Hospital Los Angeles is the highest-ranked hospital in California and fifth in the nation on the prestigious U.S. News & World Report Honor Roll of best children's hospitals. U.S. News ranks Children's Hospital Los Angeles in all 10 specialty categories. Clinical care at the hospital is led by physicians who are faculty members of the Keck School of Medicine of USC through an affiliation dating from 1932. The hospital also leads the largest pediatric residency training program at a freestanding children's hospital in the Western United States. The Saban Research Institute of Children's Hospital Los Angeles is home to all basic, translational, clinical and community research conducted at the hospital, allowing proven discoveries to quickly reach patients. Our mission: to create hope and build healthier futures. To learn more, follow us on Facebook, Instagram, LinkedIn, YouTube and Twitter, and visit our blog at CHLA.org/blog.

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