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$14M NIH grant funds gene-editing research for rare metabolic diseases at Penn and CHOP
Researchers aim to develop personalized therapies for urea cycle disorders and other genetic conditions using advanced CRISPR technology.
Children’s Hospital of Philadelphia Researchers Report Encouraging First Evidence of Effective New Gene Therapy to Treat Multiple Sulfatase Deficiency
The ex vivo gene therapy improved sulfatase production and reduced symptoms associated with the disease in preclinical models
CHOP, Penn researchers develop gene editing approaches for PKU treatment
The two studies, presented at ASHG 2023, identified base editing and prime editing approaches for treating the rare newborn genetic disease
NIH Awards Penn Medicine and Children’s Hospital of Philadelphia $26 Million Grant to Develop Therapies for Rare Newborn Genetic Diseases
A Penn Medicine and CHOP team will seek to develop treatments for three rare, incurable genetic diseases with the help of a $26 million grant from the National Institutes of Health.
CHOP Researchers Selected for Bespoke Gene Therapy Consortium
CHOP researchers will develop gene therapies for multiple sulfatase deficiency, a devastating lysosomal storage disorder, with the goal of first-in-human trials.
CHOP Nurse Practitioner Develops Improved Device for Tube-Fed Children
The device is a better way to remove excess air in the patient’s stomach, making the tube-feeding process more efficient and comfortable.
Children’s Hospital of Philadelphia Dietitian Creates Blenderized Tube Feed Recipe Builder to Help Families with Tube-Fed Children Provide Real Food, Meet Nutritional Needs
CHOP collaboration with Nestlé Health Science elevates an idea that is a win for families.
CHOP Expert in Metabolic Diseases Appointed to State Rare Diseases Council
Can Ficicioglu, MD, PhD, a physician-scientist with expertise in genetic disorders, was appointed to a statewide Rare Diseases Advisory Council by the PA Department of Health.
Turning Health Improvement Ideas into Marketable Products and Services
Children’s Hospital's Office of Entrepreneurship and Innovation empowers employees to find “a better way” and foster an entrepreneurial spirit.
CHOP Announces Membership in PLEXUS Shared Workspace
The Office of Entrepreneurship and Innovation recently announced its membership in a new shared workspace, PLEXUS, specifically designed for healthcare innovation companies.