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Translational Read-Through Therapy of RPGR Nonsense Mutations
X-chromosomal retinitis pigmentosa (RP) frequently is caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. We evaluated the potential of PTC124 (Ataluren, Translama(TM)) treatment to promote ribosomal read-through of premature termination codons (PTC) in RPGR. Expression con...
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| Udgivet i: | Int J Mol Sci |
|---|---|
| Main Authors: | , , , , , |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
MDPI
2020
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7697989/ https://ncbi.nlm.nih.gov/pubmed/33182541 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms21228418 |
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