Gene Therapy for Inherited Metabolic Disorders Program

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The Gene Therapy for Inherited Metabolic Disorders (GTIMD) Program aims to advance clinical care for patients with inborn errors of metabolism (IEMs) who received molecular therapies such as gene, mRNA, and anti-sense oligonucleotide therapies. With trials underway for molecular therapeutics in more than 15 different IEMs, an increasing number of our patients will receive a novel therapy, either in a clinical trial or, eventually, as part of routine clinical care.

The GTIMD team will educate the next generation of experts in molecular therapies for IEMs, while optimizing a roadmap to advance preclinical molecular therapies at Children's Hospital of Philadelphia and the University of Pennsylvania into the clinic through two programs:

  • Gene editing therapy for phenylketonuria
  • Ex vivo gene therapy with stem cell transplant for multiple sulfatase deficiency

Our work will expand the clinical program at Children's Hospital of Philadelphia with the goal of becoming the destination for patients worldwide to receive the latest evidence-informed care.

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