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Gene-targeted therapy for neurofibromatosis and schwannomatosis: The path to clinical trials

8 Nov 2023

Abstract excerpt

Numerous successful gene-targeted therapies are arising for the treatment of a variety of rare diseases. At the same time, current treatment options for neurofibromatosis 1 and schwannomatosis are limited and do not directly address loss of gene/protein function. In addition, treatments have mostly focused on symptomatic tumors, but have failed to address multisystem involvement in these conditions. Gene-targeted...

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PMID: 37937606
DOI: 10.1177/17407745231207970

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