Personalized allele-specific antisense oligonucleotides for GNAO1-neurodevelopmental disorder

GNAO1-associated disorders are ultra-rare autosomal dominant conditions, which can manifest, depending on the exact pathogenic variant in GNAO1, as a spectrum of neurological phenotypes, including epileptic encephalopathy, developmental delay with movement disorders, or late-onset dystonia. There ar...

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發表在:	Molecular Therapy: Nucleic Acids
Main Authors:	Inna Shomer, Nofar Mor, Shaul Raviv, Noga Budick-Harmelin, Tanya Matchevich, Sharon Avkin-Nachum, Yoach Rais, Rebecca Haffner-Krausz, Ariela Haimovich, Aviv Ziv, Reut Fluss, Bruria Ben-Ze’ev, Gali Heimer, Denis N. Silachev, Vladimir L. Katanaev, Dan Dominissini
格式:	Article
語言:	英语
出版:	Elsevier 2025-03-01
主題:	MT: Oligonucleotides: Therapies and Applications GNAO1 personalized ASOs antisense oligonucleotides ASOs individualized ASOs
在線閱讀:	http://www.sciencedirect.com/science/article/pii/S2162253124003196