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Gene, Stem Cell, and Future Therapies for Orphan Diseases

27 Jun 2012

Abstract excerpt

There are an estimated 7,000 "orphan diseases," but treatments are currently available for only about 5% of them. Recent progress in the advanced platforms of gene therapy, stem cell therapy, gene modification, and gene correction offers possibilities for new therapies and cures for rare diseases. Many rare diseases are genetic in origin, and gene therapy is being successfully applied to treat them. Human stem...

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PMID: 22739143
DOI: 10.1038/clpt.2012.82

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