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Preclinical translation of <i>Neurofibromatosis type 1</i> ( <i>NF1)</i> exon 17 skipping using targeted U7-SnRNA packaged into engineered AAV serotypes

2026-07-02

Abstract excerpt

<h4>ABSTRACT</h4> To facilitate the translation of NF1 exon 17 skipping as a mutation-specific therapy for Neurofibromatosis type 1 into in vivo testing, we have continued to develop more efficient antisense oligonucleotides (ASOs), humanized mouse models, and explored multiple delivery platforms including an adeno-associated virus (AAV)-U7-SnRNA vector approach. We evaluated both biodistribution and exon skipp...

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Literature Corpus work
395339c3-5a72-585a-8993-bbff3b13c615
DOI
10.64898/2026.06.29.734312
Open publication

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