Molecular Therapy: Nucleic Acids

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22.01.2024

Preclinical evaluation of CRISPR-based therapies for Noonan syndrome caused by deep-intronic LZTR1 variants

Authors Knauer C, Haltern H, Schoger E, Kügler S, Roos L, Zelarayán LC, Hasenfuss G, Zimmermann WH, Wollnik B, Cyganek L Journal Molecular Therapy: Nucleic Acids Citation Mol Ther Nucleic Acids. 2024 Jan 23;35(1):102123. Abstract Gene variants in LZTR1 are implicated to cause Noonan syndrome associated with a severe and
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