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

• Үндсэн зохиолчид: Irena Bočkaj (Зохиогч), Anna Moreno Garcia (Зохиогч), Pablo de Miguel Herraiz (Зохиогч), Sonay Keskin (Зохиогч), Vanessa Zancanella (Зохиогч), Şeyda Acar Broekmans (Зохиогч), Astrid Vallès (Зохиогч), Ying Poi Liu (Зохиогч), Melvin Evers (Зохиогч), Morgane Wartel (Зохиогч)
• Формат: Ном
• Хэвлэсэн: Elsevier, 2024-09-01T00:00:00Z.
• Нөхцлүүд: article
• Онлайн хандалт: 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...