CVI News: Drs. William Peranteau and Kiran Musunuru receive $26.5 Million NIH NINDS U19 Grant

William Peranteau, MD, assistant professor of Surgery at Children's Hospital of Philadelphia and Kiran Musunuru, MD, PhD, MPH, ML, professor of medicine at the University of Pennsylvania, were awarded a $26.5 Million U19 grant from the NIH National Institute of Neurological Disorders and Stroke (NINDS) for their project "Postnatal and Prenatal Therapeutic Base Editing for Metabolic Diseases." Phenylketonuria (PKU), hereditary tyrosinemia type 1 (HT1), and mucopolysaccharidosis type 1 (MPSI) are inherited disorders of metabolism that cause debilitating and even life-threatening medical problems as early as birth.

The grant will support the development of a type of CRISPR gene editing therapy, called base editing, to directly and permanently correct gene mutations or otherwise alter genes at the DNA level in the liver, either after or before birth—a “one-and-done” treatment strategy that could substantially improve the long-term health of patients with these diseases.

Kiran Musunuru, MD, PhD, MPH, ML