Work

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

Year: 2026

Type: preprint

Source: bioRxiv (Cold Spring Harbor Laboratory)

DOI: 10.64898/2026.06.29.734312

Authors: Marc Moore, Kimia Rayat-Sanati, Xiaoxia Zhang, Hui Liu, Zakaria Rostamitehrani, Tarah Vijayasarathy, Erik Westin, Miguel Sena-Esteves, Casey A. Maguire, Robert A. Kesterson, Linda Popplewell, Deeann Wallis

Institutions: Royal Holloway University of London, Teesside University, University of Alabama at Birmingham, Pennington Biomedical Research Center, University of Massachusetts Chan Medical School, Harvard University, Massachusetts General Hospital

Language: English

Topic: Virus-based gene therapy research

Subfield: Genetics

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: closed

Funder: Gilbert Family Foundation

Award: G7175014489

OpenAlex ID: W7167277516

Cites: 49

Cited by:

Related to: 10