Small Molecules Restore Bestrophin 1 Expression and Function of Both Dominant and Recessive Bestrophinopathies in Patient-Derived Retinal Pigment Epithelium
Investigative Ophthalmology & Visual Science2020Vol. 61(5), pp. 28–28
Citations Over TimeTop 23% of 2020 papers
Jingshu Liu, Rachel L. Taylor, Richard A. Baines, Lisa Swanton, Sally Freeman, Barbara Corneo, Achchhe Patel, Alan D. Marmorstein, Travis Knudsen, Graeme C. Black, Forbes Manson
Abstract
The restoration of bestrophin 1 function in patient-derived RPE confirms the US Food and Drug Administration-approved drug 4PBA as a promising therapeutic treatment for bestrophinopathies.
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