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Three‐Year Safety Results of SAR422459 (EIAV‐ABCA4) Gene Therapy in Patients With ABCA4‐Associated Stargardt Disease: An Open‐Label Dose‐Escalation Phase I/IIa Clinical Trial, Cohorts 1‐5

Maria A. Parker, Laura Erker, Isabelle Audo, Dongseok Choi, Saddek Mohand‐Saïd, Kąstytis Šeštakauskas, Patrick Benoit, Terence Appelqvist, Melissa R. Krahmer, Caroline Ségaut-Prévost, Brandon J. Lujan, Ambar Faridi, Elvira N. Chegarnov, Peter Steinkamp, Cristy A. Ku, Mariana Matioli da Palma, Pierre‐Olivier Barale, Sarah Ayelo-Scheer, Andreas Lauer, Tim Stout, David J. Wilson, Richard G. Weleber, Mark E. Pennesi, José‐Alain Sahel, Paul Yang

2022American Journal of Ophthalmology56 citationsDOIOpen Access PDF

Topics & Concepts

MedicineStargardt diseaseOphthalmologyABCA4Visual acuityChoroideremiaAdverse effectFundus photographyVisual fieldRetinal pigment epitheliumFundus (uterus)Macular degenerationRetinopathyFluorescein angiographyRetinalInternal medicineDiabetes mellitusGeneChemistryBiochemistryPhenotypeEndocrinologyRetinal Development and DisordersOcular Disorders and TreatmentsVirus-based gene therapy research
Three‐Year Safety Results of SAR422459 (EIAV‐ABCA4) Gene Therapy in Patients With ABCA4‐Associated Stargardt Disease: An Open‐Label Dose‐Escalation Phase I/IIa Clinical Trial, Cohorts 1‐5 | Litcius