Feasibility of Using NF1-GRD and AAV for Gene Replacement Therapy in NF1-Associated Tumors
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DOI: 10.1038/s41434-019-0080-9
Abstract: Neurofibromatosis type 1, including the highly aggressive malignant peripheral nerve sheath tumors (MPNSTs), is featured by the loss of functional neurofibromin 1 (NF1) protein resulting from genetic alterations. A major function of NF1 is suppressing… read more here.
Keywords: replacement therapy; grd; gene replacement; therapy nf1 ... See more keywords