Work

Rapid kinetics of AAV8 delivery of Vglut3 and hearing rescue shown day-by-day via matched IHC and ABR

Year: 2025

Type: article

Abstract: The race to implement effective gene therapy to restore hearing and sight has relied primarily on adeno-associated virus (AAV)-based vectors, due to their wide range of cell tropisms, ease of modification, and their relatively innocuous safety profile in patients. Delivery to the inner ear is non-trivial; researchers have developed multiple injection routes to reach the cochlea in animal models, which is nestled deep within the temporal bone of the skull. Newborn mice developmentally resemble a human fetus in the final month of gestation, with the eyelids and ears not fully developed.

Source: Molecular Therapy — Methods & Clinical Development

DOI: 10.1016/j.omtm.2025.101638

Authors: Cole W.D. Peters, Killian Hanlon

Institution: University of California, Los Angeles

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