Children’s Hospital Los Angeles Investigators Secure $4m for Regenerative Research & Cell Therapeutics

Children’s Hospital Los Angeles Investigators Secure $4m for Regenerative Research & Cell Therapeutics

Two prominent researchers from the Saban Research Institute of Children’s Hospital Los Angeles haves secured over $4 million from the National Institute of Diabetes and Digestive and Kidney Diseases to study cellular damage and repair in both chronic kidney disease and Alport syndrome, conditions that adversely impact the body’s ability to properly filter blood. With this award, Laura Perin, PhD, and Stefano Da Sacco, PhD, experts focusing on how disease impacts kidney cells at the molecular level, can progress this critical line of regenerative medicinal research out of Children’s Hospital Los Angeles.

Breakthrough Model

The Los Angeles, California-based team has developed a breakthrough model called the “glomerulus on a chip” to advance investigation into the mechanism of autoimmune diseases of primary membranous neuropathy. These diseases damage the cells that constitute the kidney’s filtration barrier. Dr. Da Sacco’s studies are focused on determining which molecules inside these cells are activated during injury. This step must be understood thoroughly in anticipation for the eventual development of medications and treatments for diseases such as primary membranous neuropathy.

Human Extracellular Vesicles (EVs)

In parallel, Dr. Perin investigates human extracellular vesicles (EVs), the small particles that cells release to communicate with one another and repair damage in nearby cells. Thus far, Dr. Perin has demonstrated that EVs offer options for protection in a preclinical model for Alport syndrome, which causes irreversible damage to the cells that make up the kidney’s filtration barrier.

Regenerative Research & Cell Therapeutics

Both Dr. Perin and Dr. Da Sacco work in the Children’s Hospital of Los Angeles GOFARR Laboratory for Organ Regenerative Research and Cell Therapeutics in Urology, emphasizing tissue engineering, cell therapies, and the discovery of new treatments for kidney disease. Their work is supported by multiple core facilities at CHLA, including the Extracellular Vesicle Core, offering expertise and leading-edge technologies to support EV research.  Based on this cutting-edge research, Children’s Hospital Los Angeles emerges as a leader in regenerative research and cell therapeutics.

Grant Awards

Dr. Da Sacco was been awarded a $1.9 million 5-year grant (1R01DK123234-01A1) while Dr. Perin’s award totals $2.2 million over 4 years. (1R01DK121037-01A1).

GOFARR Laboratory

GOFARR Laboratory for Organ Regenerative Research & Cell Therapeutics in Urology is led by Roger De Filippo, MD, and Laura Perin, PhD, both serving as principal investigators and co-directors. The focus of this research group is the development of new stem cell therapeutics and other techniques for organ regeneration.

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 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.

Lead Research/Investigator

Laura Perin, PhD

Stefano Da Sacco, PhD