Toward an all-in-one recombinant adeno-associated virus vector for functionally ablating the prion gene using CRISPR-Cas technology.

Any strategy that can selectively and persistently lower the brain levels of the cellular prion protein (PrPC) is expected to extend survival in prion diseases. Recent advances in the virus-mediated delivery of gene therapies prompted us to explore if a recombinant adeno-associated virus (rAAV) vect...

Ausführliche Beschreibung

Bibliografische Detailangaben

Hauptverfasser:	Claire Verkuyl, Ari Belotserkovsky, Thomas Zerbes, Declan Williams, Medha R Krishnan, Sabrina Zhu, Sophie Grunnesjӧ, Shehab Eid, Cunjie Zhang, Wenda Zhao, Leo Xu, Eleanore Lin, Teaghan O'Shea, Benjamin Draper, Andreas Jungman, Patrick Most, Gerold Schmitt-Ulms
Format:	Artigo
Sprache:	Inglês
Veröffentlicht:	Public Library of Science (PLoS) 2025-01-01
Schriftenreihe:	PLoS ONE
Online-Zugang:	https://doi.org/10.1371/journal.pone.0336578
Tags:	Tag hinzufügen Keine Tags, Fügen Sie das erste Tag hinzu!