Article

Proof-of-principle of NF1 Gene Therapy in plexiform neurofibroma mice models

2025-01-24

Abstract excerpt

<h4>ABSTRACT</h4> Neurofibromatosis type I is a rare neurocutaneous syndrome characterized by the development of disfiguring neurofibroma tumors with unmet clinical needs. As Neurofibromatosis Type I is a monogenic disease, the development of gene therapy is highly attractive, but it is currently unknown if rescuing the NF1 gene in established neurofibroma is sufficient for tumor regression. Here, we test this h...

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Literature Corpus work: 43145e1a-c055-5763-b8ff-e4cf4b55151e
DOI: 10.1101/2025.01.21.634081

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