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Testing the efficacy of new NF1 gene therapy candidates in human cell types

While intervention approaches range from NF1 gene replacement to targeting a genetic factor in the Ras signaling pathway are promising, there is currently no humanized …
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Optimized AAV vectors for therapeutic Schwann cell targeting in NF1

There is an urgent need to identify new treatments to prevent or shrink PNs. Targeted gene therapies that use viruses to restore the function of …
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Nanoparticle Delivery of Therapeutic NF1 Signaling ERPs to Schwann Cells

Recent advances in genome editing provide an opportunity to therapeutically address loss of the tumor suppressor gene neurofibromin 1 (NF1) and prevent neurofibromatosis type 1 …
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