LinQURE: A novel AAV gene silencing platform that supports multi-transcript targeting for complex disorders
Irena Bočkaj, Anna Moreno Garcia, Pablo de Miguel Herraiz, Sonay Keskin et autres
, 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.
nl (code pays fourni par la source)
0 citations
Molecular Therapy — Nucleic Acids