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

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

Gene variants in LZTR1 are implicated to cause Noonan syndrome associated with a severe and early-onset hypertrophic cardiomyopathy. Mechanistically, LZTR1 deficiency results in accumulation of RAS GTPases and, as a consequence, in RAS-MAPK signaling hyperactivity, thereby causing the Noonan syndrom...

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Bibliographic Details
Main Authors: Carolin Knauer, Henrike Haltern, Eric Schoger, Sebastian Kügler, Lennart Roos, Laura C. Zelarayán, Gerd Hasenfuss, Wolfram-Hubertus Zimmermann, Bernd Wollnik, Lukas Cyganek
Format: Article
Language:English
Published: Elsevier 2024-03-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: RNA/DNA Editing
cardiomyocytes
CRISPR-Cas9
gene therapy
genome editing
hypertrophic cardiomyopathy
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253124000106

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http://www.sciencedirect.com/science/article/pii/S2162253124000106

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