Research ArticleGene Therapy
Plasticity of the human visual system after retinal gene therapy in patients with Leber’s congenital amaurosis
- Manzar Ashtari1,2,*,
- Hui Zhang3,
- Philip A. Cook4,
- Laura L. Cyckowski5,
- Kenneth S. Shindler1,2,
- Kathleen A. Marshall6,
- Puya Aravand1,2,
- Arastoo Vossough5,
- James C. Gee4,
- Albert M. Maguire1,2,6,
- Chris I. Baker7 and
- Jean Bennett1,2,6
- 1Center for Advanced Retinal and Ocular Therapeutics, University of Pennsylvania Perelman School of Medicine, 309 Stellar-Chance Labs, 422 Curie Boulevard, Philadelphia, PA 19104, USA.
- 2F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA 19014, USA.
- 3Department of Computer Science and Centre for Medical Image Computing, University College London, Gower Street, London WC1E 6BT, UK.
- 4Penn Image Computing and Science Laboratory, Department of Radiology, University of Pennsylvania, 3600 Market Street, Philadelphia, PA 19104, USA.
- 5Department of Radiology, The Children’s Hospital of Philadelphia, Philadelphia, PA 19014, USA.
- 6Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia, Colket Translational Research Building, 3501 Civic Center Boulevard, Philadelphia, PA 19014, USA.
- 7Laboratory of Brain and Cognition, National Institutes of Health, 10 Center Drive, MSC 1240, Bethesda, MD 20892, USA.
- ↵*Corresponding author. E-mail: ashtari{at}mail.med.upenn.edu
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Science Translational Medicine 15 Jul 2015:
Vol. 7, Issue 296, pp. 296ra110
DOI: 10.1126/scitranslmed.aaa8791
Vol. 7, Issue 296, pp. 296ra110
DOI: 10.1126/scitranslmed.aaa8791
Manzar Ashtari
1Center for Advanced Retinal and Ocular Therapeutics, University of Pennsylvania Perelman School of Medicine, 309 Stellar-Chance Labs, 422 Curie Boulevard, Philadelphia, PA 19104, USA.
2F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA 19014, USA.
Hui Zhang
3Department of Computer Science and Centre for Medical Image Computing, University College London, Gower Street, London WC1E 6BT, UK.
Philip A. Cook
4Penn Image Computing and Science Laboratory, Department of Radiology, University of Pennsylvania, 3600 Market Street, Philadelphia, PA 19104, USA.
Laura L. Cyckowski
5Department of Radiology, The Children’s Hospital of Philadelphia, Philadelphia, PA 19014, USA.
Kenneth S. Shindler
1Center for Advanced Retinal and Ocular Therapeutics, University of Pennsylvania Perelman School of Medicine, 309 Stellar-Chance Labs, 422 Curie Boulevard, Philadelphia, PA 19104, USA.
2F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA 19014, USA.
Kathleen A. Marshall
6Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia, Colket Translational Research Building, 3501 Civic Center Boulevard, Philadelphia, PA 19014, USA.
Puya Aravand
1Center for Advanced Retinal and Ocular Therapeutics, University of Pennsylvania Perelman School of Medicine, 309 Stellar-Chance Labs, 422 Curie Boulevard, Philadelphia, PA 19104, USA.
2F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA 19014, USA.
Arastoo Vossough
5Department of Radiology, The Children’s Hospital of Philadelphia, Philadelphia, PA 19014, USA.
James C. Gee
4Penn Image Computing and Science Laboratory, Department of Radiology, University of Pennsylvania, 3600 Market Street, Philadelphia, PA 19104, USA.
Albert M. Maguire
1Center for Advanced Retinal and Ocular Therapeutics, University of Pennsylvania Perelman School of Medicine, 309 Stellar-Chance Labs, 422 Curie Boulevard, Philadelphia, PA 19104, USA.
2F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA 19014, USA.
6Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia, Colket Translational Research Building, 3501 Civic Center Boulevard, Philadelphia, PA 19014, USA.
Chris I. Baker
7Laboratory of Brain and Cognition, National Institutes of Health, 10 Center Drive, MSC 1240, Bethesda, MD 20892, USA.
Jean Bennett
1Center for Advanced Retinal and Ocular Therapeutics, University of Pennsylvania Perelman School of Medicine, 309 Stellar-Chance Labs, 422 Curie Boulevard, Philadelphia, PA 19104, USA.
2F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA 19014, USA.
6Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia, Colket Translational Research Building, 3501 Civic Center Boulevard, Philadelphia, PA 19014, USA.
