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Gene Therapy Reforms Photoreceptor Structure and Restores Vision in NPHP5-associated Leber Congenital Amaurosis
Gustavo D. Aguirre, Artur V. Cideciyan, Valérie L. Dufour, Ana Ripolles García, Raghavi Sudharsan, Malgorzata Swider, Roman Nikonov, Simone Iwabe, Sanford L. Boye, William W. Hauswirth, Samuel G. Jacobson, William A. Beltran
doi: https://doi.org/10.1101/2020.10.07.329821
Gustavo D. Aguirre
aDivision of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104
Artur V. Cideciyan
bScheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104
Valérie L. Dufour
aDivision of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104
Ana Ripolles García
aDivision of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104
Raghavi Sudharsan
aDivision of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104
Malgorzata Swider
bScheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104
Roman Nikonov
aDivision of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104
Simone Iwabe
aDivision of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104
Sanford L. Boye
cPowell Gene Therapy Center, Department of Pediatrics, University of Florida, Gainesville, FL 32610
William W. Hauswirth
dDepartment of Ophthalmology, University of Florida, Gainesville, FL 32610
Samuel G. Jacobson
bScheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104
William A. Beltran
aDivision of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104
Posted October 08, 2020.
Gene Therapy Reforms Photoreceptor Structure and Restores Vision in NPHP5-associated Leber Congenital Amaurosis
Gustavo D. Aguirre, Artur V. Cideciyan, Valérie L. Dufour, Ana Ripolles García, Raghavi Sudharsan, Malgorzata Swider, Roman Nikonov, Simone Iwabe, Sanford L. Boye, William W. Hauswirth, Samuel G. Jacobson, William A. Beltran
bioRxiv 2020.10.07.329821; doi: https://doi.org/10.1101/2020.10.07.329821
Gene Therapy Reforms Photoreceptor Structure and Restores Vision in NPHP5-associated Leber Congenital Amaurosis
Gustavo D. Aguirre, Artur V. Cideciyan, Valérie L. Dufour, Ana Ripolles García, Raghavi Sudharsan, Malgorzata Swider, Roman Nikonov, Simone Iwabe, Sanford L. Boye, William W. Hauswirth, Samuel G. Jacobson, William A. Beltran
bioRxiv 2020.10.07.329821; doi: https://doi.org/10.1101/2020.10.07.329821
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