In Vivo Efficacy and Safety Evaluations of Therapeutic Splicing Correction Using U1 snRNA in the Mouse Retina

In Vivo Efficacy and Safety Evaluations of Therapeutic Splicing Correction Using U1 snRNA in the Mouse Retina

Efficacy and safety considerations constitute essential steps during development of in vivo gene therapies. Herein, we evaluated efficacy and safety of splice factor-based treatments to correct mutation-induced splice defects in an <i>Opa1</i> mutant mouse line. We applied adeno-associat...

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Bibliographic Details
Main Authors: Sebastian Swirski, Oliver May, Malte Ahlers, Bernd Wissinger, Martin Greschner, Christoph Jüschke, John Neidhardt
Format: Article
Language:English
Published: MDPI AG 2023-03-01
Series:Cells
Subjects:
subretinal injection
gene therapy
U1 snRNA
U6 snRNA
AAV
splice correction
Online Access:https://www.mdpi.com/2073-4409/12/6/955

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