Work

LinQURE: A novel AAV gene silencing platform that supports multi-transcript targeting for complex disorders

Year: 2024

Type: article

Abstract: , allowing more efficient downregulation of their disease-causing mRNA targets. This approach supports the development of multi-targeting therapeutic strategies, enabling gene therapy products to adapt to more complex multigenic indications, thus expanding the toolbox of readily available gene therapies.

Source: Molecular Therapy — Nucleic Acids

DOI: 10.1016/j.omtn.2024.102307