Interim safety and efficacy of gene therapy for RLBP1-associated retinal dystrophy: a phase 1/2 trial.

Autor: Kvanta A; Department of Clinical Neuroscience, St. Erik Eye Hospital, Karolinska Institutet, Stockholm, Sweden. anders.kvanta@ki.se., Rangaswamy N; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Holopigian K; Novartis Institutes for Biomedical Research, East Hanover, NJ, USA., Watters C; Novartis Pharmaceuticals Corporation, Cambridge, MA, USA., Jennings N; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Liew MSH; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Bigelow C; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Grosskreutz C; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Burstedt M; Department of Clinical Sciences/Ophthalmology, University of Umeå, Umeå, Sweden., Venkataraman A; Department of Clinical Neuroscience, St. Erik Eye Hospital, Karolinska Institutet, Stockholm, Sweden., Westman S; Department of Clinical Neuroscience, St. Erik Eye Hospital, Karolinska Institutet, Stockholm, Sweden., Geirsdottir A; Department of Clinical Neuroscience, St. Erik Eye Hospital, Karolinska Institutet, Stockholm, Sweden., Stasi K; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., André H; Department of Clinical Neuroscience, St. Erik Eye Hospital, Karolinska Institutet, Stockholm, Sweden.

Publikováno v: Nature communications [Nat Commun] 2024 Sep 10; Vol. 15 (1), pp. 7438. Date of Electronic Publication: 2024 Sep 10.