RNAi-mediated rheostat for dynamic control of AAV-delivered transgenes

Adeno-associated virus (AAV)-based gene therapy could be facilitated by the development of molecular switches to control the magnitude and timing of expression of therapeutic transgenes. RNA interference (RNAi)-based approaches hold unique potential as a clinically proven modality to pharmacological...

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Detalles Bibliográficos
Autores principales: Subramanian, Megha, McIninch, James, Zlatev, Ivan, Schlegel, Mark K., Kaittanis, Charalambos, Nguyen, Tuyen, Agarwal, Saket, Racie, Timothy, Alvarado, Martha Arbaiza, Wassarman, Kelly, Collins, Thomas S., Chickering, Tyler, Brown, Christopher R., Schmidt, Karyn, Castoreno, Adam B., Shulga-Morskaya, Svetlana, Stamenova, Elena, Buckowing, Kira, Berman, Daniel, Barry, Joseph D., Bisbe, Anna, Maier, Martin A., Fitzgerald, Kevin, Jadhav, Vasant
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10082758/
https://www.ncbi.nlm.nih.gov/pubmed/37031257
http://dx.doi.org/10.1038/s41467-023-37774-5

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