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Research ArticleGene Therapy
AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness
- Jean Bennett1,2,*,†,
- Manzar Ashtari3,*,†,
- Jennifer Wellman2,
- Kathleen A. Marshall2,
- Laura L. Cyckowski3,
- Daniel C. Chung1,2,
- Sarah McCague2,
- Eric A. Pierce1,4,‡,
- Yifeng Chen2,
- Jeannette L. Bennicelli1,
- Xiaosong Zhu4,
- Gui-shuang Ying5,
- Junwei Sun2,
- J. Fraser Wright2,
- Alberto Auricchio6,7,
- Francesca Simonelli6,8,
- Kenneth S. Shindler1,
- Federico Mingozzi2,
- Katherine A. High2,9 and
- Albert M. Maguire1,2,4
- 1F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, 309 Stellar-Chance Labs, 422 Curie Boulevard, Philadelphia, PA 19104, USA.
- 2Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia, Colket Translational Research Building, 3501 Civic Center Boulevard, Philadelphia, PA 19014, USA.
- 3Department of Radiology, The Children’s Hospital of Philadelphia, Philadelphia, PA 19014, USA.
- 4Department of Ophthalmology, Children’s Hospital of Philadelphia, Philadelphia, PA 19014, USA.
- 5Scheie Eye Institute, Center for Preventive Ophthalmology and Biostatistics, Department of Ophthalmology, University of Pennsylvania, 3535 Market Street, Suite 700, Philadelphia, PA 19104, USA.
- 6Telethon Institute of Genetics and Medicine, Via P. Castellino 111, 80131 Naples, Italy.
- 7Medical Genetics, Department of Pediatrics, “Federico II” University, Via S. Pansini 5, 80131 Naples, Italy.
- 8Department of Ophthalmology, Seconda Università degli Studi di Napoli, Via S. Pansini 5, 80131 Naples, Italy.
- 9Howard Hughes Medical Institute, 3615 Civic Center Boulevard, Philadelphia, PA 19104, USA.
- ↵†To whom correspondence should be addressed. E-mail: jebennet{at}mail.med.upenn.edu (J.B.); ashtari{at}email.chop.edu (M.A.)
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Science Translational Medicine 08 Feb 2012:
Vol. 4, Issue 120, pp. 120ra15
DOI: 10.1126/scitranslmed.3002865
Vol. 4, Issue 120, pp. 120ra15
DOI: 10.1126/scitranslmed.3002865
Jean Bennett
Manzar Ashtari
Jennifer Wellman
Kathleen A. Marshall
Laura L. Cyckowski
Daniel C. Chung
Sarah McCague
Eric A. Pierce
Yifeng Chen
Jeannette L. Bennicelli
Xiaosong Zhu
Gui-shuang Ying
Junwei Sun
J. Fraser Wright
Alberto Auricchio
Francesca Simonelli
Kenneth S. Shindler
Federico Mingozzi
Katherine A. High
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