The goal of medicine is to alleviate needless suffering from injury and disease. Even
though human affliction is no different whether brought about by rare or by widespread
disease, the attention of experts and societal commitment may differ. Thus, it is
important to stress that gene therapy approaches are applicable both to orphan diseases
and to the diseases that cause much of the morbidity and mortality in the world.
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Published online: February 18, 2013
Footnotes
Conflict of interest: None.
Supported by grants from the National Institutes of Health, Department of Defense, DebRA International, Jackson Gabriel Silver Fund, Epidermolysis Bullosa Medical Research Fund, University of Pennsylvania grant MPS I-11-009-01, and Children’s Cancer Research Fund, Minnesota.
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© 2013 Mosby, Inc. Published by Elsevier Inc. All rights reserved.
