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September 29, 2014

Gene Therapy Targeting Liver Corrects Cardiovascular Symptoms in Animal Model of Rare Enzyme Deficiency Disease

In the second of two papers outlining new gene-therapy approaches to treat a rare disease called MPS I, researchers from Perelman School of Medicine at the University of Pennsylvania examined systemic delivery of a vector to replace the enzyme IDUA, which is deficient in patients with this disorder. Read more ...

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Penn Medicine Magazine

Spring 2014

Current Issue: Spring 2014

Researchers in the rapidly expanding research field of microbiomics have shown that the human body is home to an entire ecosystem of bacteria, viruses, fungi, and other microbes. What's more, these microbes play an important role in regulating many of our physiological processes.

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