KJ Stepping into the Holidays After a Milestone Year
Dec 17, 2025
KJ is celebrating a big year after becoming the first patient in the world to receive a personalized CRISPR gene editing therapy to treat his rare disease.
Metabolic Disease Program News
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World's First Patient Treated with Personalized CRISPR Gene Editing Therapy at Children’s Hospital of Philadelphia
May 15, 2025
Landmark study from CHOP and Penn Medicine showcases the power of customized gene editing therapy to treat patient with rare metabolic disease.
$14M NIH grant funds gene-editing research for rare metabolic diseases at Penn and CHOP
Oct 10, 2024
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
Sep 4, 2024
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
Nov 3, 2023
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
Aug 1, 2023
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
May 24, 2023
CHOP researchers will develop gene therapies for multiple sulfatase deficiency, a devastating lysosomal storage disorder, with the goal of first-in-human trials.