Translational Read-Through Therapy of <i>RPGR</i> Nonsense Mutations

Translational Read-Through Therapy of <i>RPGR</i> Nonsense Mutations

X-chromosomal retinitis pigmentosa (RP) frequently is caused by mutations in the retinitis pigmentosa GTPase regulator (<i>RPGR</i>) gene. We evaluated the potential of PTC124 (Ataluren, Translama<sup>TM</sup>) treatment to promote ribosomal read-through of premature terminat...

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Bibliographic Details
Main Authors: Christine Vössing, Marta Owczarek-Lipska, Kerstin Nagel-Wolfrum, Charlotte Reiff, Christoph Jüschke, John Neidhardt
Format: Article
Language:English
Published: MDPI AG 2020-11-01
Series:International Journal of Molecular Sciences
Subjects:
RPGR
transcriptional therapy
read-through therapy
cilia
cilium
treatment
Online Access:https://www.mdpi.com/1422-0067/21/22/8418

Internet

https://www.mdpi.com/1422-0067/21/22/8418

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