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

• Main Authors: Irena Bočkaj (Author), Anna Moreno Garcia (Author), Pablo de Miguel Herraiz (Author), Sonay Keskin (Author), Vanessa Zancanella (Author), Şeyda Acar Broekmans (Author), Astrid Vallès (Author), Ying Poi Liu (Author), Melvin Evers (Author), Morgane Wartel (Author)
• Format: Book
• Published: Elsevier, 2024-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Given that numerous genetic disorders, driven by diverse pathogenic mechanisms, may be amenable to recombinant adeno-associated virus (rAAV)-delivered gene therapies, the sustained innovation of rAAV-based therapeutic modalities is crucial. The progression and severity of genetic diseases can be red...