Felix F Reichel, M Dominik Fischer, Katarina Stingl, Laura Kuehlewein, Immanuel Seitz, Tobias Peters, Melanie Ziegler, Barbara Wilhelm, Susanne Kohl, Nicole Weisschuh, Peter Martus, Mathias Seeliger, Regine Muehlfriedel, Francois Paquet-Durand, Stephen H Tsang, Karl Ulrich Bartz-Schmidt, Marius Ueffing, Eberhart Zrenner, Martin Biel, Bernd Wissinger, Stylianos Michalakis, RD-Cure Consortium
چکیده
PDE6A-associated retinitis pigmentosa (RP) is a rare inherited retinal disease leading to severe vision loss and blindness, with no available treatment. This study assessed the safety and vision outcomes of a gene therapy using an adeno-associated virus (AAV) vector encoding PDE6A (AAV8.hPDE6A). In…
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DOI: 10.1136/bjo-2024-326480