Work

High-potency MyoAAV capsids enhanced skeletal muscle correction in a mouse model of GSD IIIa

Year: 2025

Type: article

Abstract: vg/kg). Validation in human liver chimeric mice revealed that the MyoAAV vectors and the AAV9 vectors had a similar efficiency in transducing human hepatocytes, indicating increased translatability for clinical applications.

Source: Molecular Therapy — Methods & Clinical Development

DOI: 10.1016/j.omtm.2025.101567

Authors: Kuo-An Liao, Sang-oh Han, Mercedes Barzi, Haiqing Yi, William Eisner, Beatrice Bissig-Choisat, Karl-Dimiter Bissig, Baodong Sun

Institutions: Duke University, Duke Medical Center

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: gold

Estimated APC paid: $3,900

Funders: Duke University, National Institute of Diabetes and Digestive and Kidney Diseases, National Institute of Arthritis and Musculoskeletal and Skin Diseases

Awards: G2911790882, G8939479389, Gene therapy for glycogen storage disease type III, Dissecting AAV silencing in humanized mice

OpenAlex ID: W4413302951

FWCI: 1.066

Cites: 34

Cited by: 2

Related to: 10