Journal
COLD SPRING HARBOR PERSPECTIVES IN MEDICINE
Volume 4, Issue 4, Pages -Publisher
COLD SPRING HARBOR LAB PRESS, PUBLICATIONS DEPT
DOI: 10.1101/cshperspect.a015149
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Funding
- National Institute of Arthritis and Musculoskeletal and Skin Diseases
- National Institute of Health [R01 AR055914]
- California Institute for Regenerative Medicine [DR1-01454]
- Epidermolysis Bullosa Medical Research Foundation
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The skin possesses qualities that make it desirable for gene therapy, and studies have focused on gene therapy for multiple cutaneous diseases. Gene therapy uses a vector to introduce genetic material into cells to alter gene expression, negating a pathological process. This can be accomplished with a variety of viral vectors or nonviral administrations. Although results are promising, there are several potential pitfalls that must be addressed to improve the safety profile to make gene therapy widely available clinically.
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