PO
Paul Orchard
Principal Investigator
University of Minnesota
I'm working on
7 months ago
Development of Lentiviral Gene Therapy for Adrenoleukodystrophy
About
Recently a lentiviral based gene therapy for cerebral adrenoleukodystrophy has been licensed, and has shown some benefit. However, several patients have developed myelodysplasia following gene therapy, likely related to the vector design. We propose a system likely to provided enhanced safety.
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