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 Opa1 mutant mouse line. We applied adeno-associated viruses to the r...

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Detalles Bibliográficos
Autores principales: Swirski, Sebastian, May, Oliver, Ahlers, Malte, Wissinger, Bernd, Greschner, Martin, Jüschke, Christoph, Neidhardt, John
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10047704/
https://www.ncbi.nlm.nih.gov/pubmed/36980294
http://dx.doi.org/10.3390/cells12060955

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10047704/
https://www.ncbi.nlm.nih.gov/pubmed/36980294
http://dx.doi.org/10.3390/cells12060955

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