Associated Papers
Translational readthrough using TRIDs - Achievements and challenges for the treatment of inherited retinal disorders.
Nagel-Wolfrum K et al.
Molecular Aspects of Medicine, 2026
Nonsense Mutations in Rare and Ultra-Rare Human Disorders: An Overview.
Vitale E et al.
Iubmb Life, 2025
Molecular Therapy for Choroideremia: Pre-clinical and Clinical Progress to Date.
Kalatzis V et al.
Molecular Diagnosis & Therapy, 2021
Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?
Han RC et al.
Expert Opinion on Orphan Drugs, 2021
Nonsense-mediated mRNA decay efficiency varies in choroideremia providing a target to boost small molecule therapeutics.
Sarkar H et al.
Human Molecular Genetics, 2019
The effect of PTC124 on choroideremia fibroblasts and iPSC-derived RPE raises considerations for therapy.
Torriano S et al.
Scientific Reports, 2018
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