OPGx-BEST1 is being developed for bestrophin-1 (BEST1)-related inherited retinal diseases or bestrophinopathies, a form of macular degeneration found primarily in adults. Results from a ...

Another gene therapy candidate, OPGx-BEST1, which Opus is developing for treatment of bestrophin-1 (BEST1)-related IRD, is on track to enter the clinic by the fourth quarter of 2025, with ...

OPGx-BEST1 is being developed for bestrophin-1 (BEST1)-related inherited retinal diseases or bestrophinopathies, a form of macular degeneration found primarily in adults. Results from a ...

Another gene therapy candidate, OPGx-BEST1, which Opus is developing for treatment of bestrophin-1 (BEST1)-related IRD, is on track to enter the clinic by the fourth quarter of 2025, with preliminary ...

A gene is a region of DNA that contains all the instructions needed to make a specific protein. If a spelling mistake (mutation) occurs in the gene DNA sequence, a faulty protein can be produced, ...

Bestrophinopathies are caused by mutations in the BEST1 gene, which contains the instructions to make the Bestrophin-1 (BEST1) protein. The BEST1 protein is produced exclusively in the RPE cells of ...

Edited by Janet R. Sparrow, Columbia University, New York, NY, and accepted by Editorial Board Member Jeremy Nathans October 13, 2018 (received for review February 9, 2018) To estimate the relative ...

Department of Biochemistry, Cellular, and Molecular Biology, University of Tennessee, Knoxville, Tennessee 37996-0840, Genome Science and Technology Program, University of Tennessee Oak Ridge National ...

Research Unit of Structural Chemistry & Computational Biophysics, Leibniz-Forschungsinstitut für Molekulare Pharmakologie, Berlin 13125, Germany ...