Article

Exon Skipping as a Therapeutic for Neurofibromatosis Type I

2021-07-26

Abstract excerpt

<title>Abstract</title> <p>We investigated the feasibility of utilizing an exon skipping approach as a genotype-dependent therapeutic for neurofibromatosis type 1 (NF1) by determining which NF1 exons might be skipped while maintaining neurofibromin function. Human neurofibromin is well-known as a GTPase activating protein (GAP), but outside of its GAP-related domain (GRD), it is unclear how critical other regions...

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Literature Corpus work: 5a3b2800-cb4a-534b-ba50-5f67662060ac
DOI: 10.21203/rs.3.rs-751331/v1

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