Sponsor: Stanford University
Researchers: M. Peter Marinkovich, MD, Jean Tang, MD, PhD
This trial will create a graft, which the investigators call “LEAES,” of the patient’s own skin that has been genetically engineered in the investigators lab to express this missing protein. The purpose of this study is to achieve proof-of-concept for this general approach to cell-based gene therapy in humans and to set the stage for further therapeutic extension in RDEB. The investigators will, in essence, take a subject’s own cells, correct them in culture, and then transplant the corrected cells back onto the subject.
Study Status: Phase 1, Active
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