CHOP, Penn researchers develop gene editing approaches for PKU treatment
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The two studies, presented at ASHG 2023, identified base editing and prime editing approaches for treating the rare newborn genetic disease
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The two studies, presented at ASHG 2023, identified base editing and prime editing approaches for treating the rare newborn genetic disease
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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.
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CHOP researchers will develop gene therapies for multiple sulfatase deficiency, a devastating lysosomal storage disorder, with the goal of first-in-human trials.
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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.