Research ArticleGene Therapy

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

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Science Translational Medicine  08 Feb 2012:
Vol. 4, Issue 120, pp. 120ra15
DOI: 10.1126/scitranslmed.3002865

Article Information

vol. 4 no. 120 120ra15

PubMed: 
Print ISSN: 
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History: 
  • Received for publication July 6, 2011
  • Accepted for publication January 19, 2012
  • .

Author Information

  1. Jean Bennett1,2,*,,
  2. Manzar Ashtari3,*,,
  3. Jennifer Wellman2,
  4. Kathleen A. Marshall2,
  5. Laura L. Cyckowski3,
  6. Daniel C. Chung1,2,
  7. Sarah McCague2,
  8. Eric A. Pierce1,4,,
  9. Yifeng Chen2,
  10. Jeannette L. Bennicelli1,
  11. Xiaosong Zhu4,
  12. Gui-shuang Ying5,
  13. Junwei Sun2,
  14. J. Fraser Wright2,
  15. Alberto Auricchio6,7,
  16. Francesca Simonelli6,8,
  17. Kenneth S. Shindler1,
  18. Federico Mingozzi2,
  19. Katherine A. High2,9 and
  20. Albert M. Maguire1,2,4
  1. 1F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, 309 Stellar-Chance Labs, 422 Curie Boulevard, Philadelphia, PA 19104, USA.
  2. 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.
  3. 3Department of Radiology, The Children’s Hospital of Philadelphia, Philadelphia, PA 19014, USA.
  4. 4Department of Ophthalmology, Children’s Hospital of Philadelphia, Philadelphia, PA 19014, USA.
  5. 5Scheie Eye Institute, Center for Preventive Ophthalmology and Biostatistics, Department of Ophthalmology, University of Pennsylvania, 3535 Market Street, Suite 700, Philadelphia, PA 19104, USA.
  6. 6Telethon Institute of Genetics and Medicine, Via P. Castellino 111, 80131 Naples, Italy.
  7. 7Medical Genetics, Department of Pediatrics, “Federico II” University, Via S. Pansini 5, 80131 Naples, Italy.
  8. 8Department of Ophthalmology, Seconda Università degli Studi di Napoli, Via S. Pansini 5, 80131 Naples, Italy.
  9. 9Howard Hughes Medical Institute, 3615 Civic Center Boulevard, Philadelphia, PA 19104, USA.
  1. To whom correspondence should be addressed. E-mail: jebennet{at}mail.med.upenn.edu (J.B.); ashtari{at}email.chop.edu (M.A.)
  • * These authors contributed equally to this work.

  • Present address: Ocular Genomics Institute, Massachusetts Eye and Ear Infirmary, Boston, MA 02114, USA.

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