Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
- Author
- MAGUIRE, Albert M1 2 3 ; SIMONELLI, Francesca4 5 ; ROSSI, Settimio4 ; LYUBARSKY, Arkady1 3 ; ARRUDA, Valder R2 3 ; KONKLE, Barbara3 ; STONE, Edwin6 7 ; JUNWEI SUN2 ; JACOBS, Jonathan8 ; DELL'OSSO, Lou8 ; HERTLE, Richard9 ; MA, Jian-Xing10 ; PIERCE, Eric A1 3 ; REDMOND, T. Michael11 ; XIAOSONG ZHU2 ; HAUCK, Bernd2 ; ZELENAIA, Olga2 ; SHINDLER, Kenneth S1 3 ; MAGUIRE, Maureen G1 3 ; FRASER WRIGHT, J2 3 ; VOLPE, Nicholas J1 3 ; MCDONNELL, Jennifer Wellman2 ; AURICCHIO, Alberto12 5 ; PUGH, Edward N1 ; HIGH, Katherine A2 3 7 ; BENNETT, Jean1 2 ; MINGOZZI, Federico2 ; BENNICELLI, Jeannette1 3 ; BANFI, Sandro5 ; MARSHALL, Kathleen A2 ; TESTA, Francesco4 ; SURACE, Enrico M5
[1] Scheie Eye Institute, University of Pennsylvania, United States
[2] Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, United States
[3] University of Pennsylvania School of Medicine, Philadelphia, United States
[4] Seconda Universita degli Studi di Napoli, Italy
[5] Telethon Institute of Genetics and Medicine, Italy
[6] the University of Iowa Carver Center, College of Medicine, Iowa City, United States
[7] Howard Hughes Medical Institute, United States
[8] Veterans Affairs Medical Center, Cleveland, United States
[9] Children's Hospital of Pittsburgh, Pittsburgh, United States
[10] University of Oklahoma, Oklahoma City, United States
[11] National Eye Institute, Bethesda, MD, United States
[12] Federico II University, Naples, Italy - Source
The New England journal of medicine. 2008, Vol 358, Num 21, pp 2240-2248, 9 p ; ref : 25 ref
- CODEN
- NEJMAG
- ISSN
- 0028-4793
- Scientific domain
- General medicine general surgery
- Publisher
- Massachusetts Medical Society, Boston, MA
- Publication country
- United States
- Document type
- Article
- Language
- English
- Keyword (fr)
- Congénital Dégénérescence neurorétinienne héréditaire de Leber Efficacité traitement Maladie congénitale Médecine Sécurité Toxicité Transfert génétique Maladie héréditaire Pathologie de l'oeil Rétinopathie
- Keyword (en)
- Congenital Leber amaurosis Treatment efficiency Congenital disease Medicine Safety Toxicity Genetic transfer Genetic disease Eye disease Retinopathy
- Keyword (es)
- Congénito Degeneración neuroretiniana hereditaria Leber Eficacia tratamiento Enfermedad congénita Medicina Seguridad Toxicidad Transferencia genética Enfermedad hereditaria Ojo patología Retinopatía
- Classification
- Pascal
- 002 Biological and medical sciences / 002B Medical sciences / 002B09 Ophthalmology / 002B09I Retinopathies
- Discipline
- Generalities in medical sciences Ophthalmology
- Origin
- Inist-CNRS
- Database
- PASCAL
- INIST identifier
- 20351675
Sauf mention contraire ci-dessus, le contenu de cette notice bibliographique peut être utilisé dans le cadre d’une licence CC BY 4.0 Inist-CNRS / Unless otherwise stated above, the content of this bibliographic record may be used under a CC BY 4.0 licence by Inist-CNRS / A menos que se haya señalado antes, el contenido de este registro bibliográfico puede ser utilizado al amparo de una licencia CC BY 4.0 Inist-CNRS
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DOI
10.1056/NEJMoa0802315 
https://dx.doi.org/10.1056/NEJMoa0802315
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