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Evaluation of rAAV2/2-ND4 Gene Therapy Efficacy in Leber Hereditary Optic Neuropathy Using an External Control Group of Untreated Patients (4108)

Year: 2021
Type: article
Study design: Clinical Trial
Source: Neurology
DOI: 10.1212/wnl.96.15_supplement.4108
Authors: Nancy Newman, Patrick Yu-Wai-Man, Valerio Carelli, Mark Moster, Catherine Vignal-Clermont, Alfredo Sadun, Chiara La Morgia, Francesco Bandello, Lorena Castillo Campillo, Stéphanie Leruez, Dean Cestari, Rod Foroozan, Rustum Karanjia, Laure Blouin, Michel Roux, Magali Taiel, Jose-Alain Sahel
Institutions: Emory University, University of Cambridge, Moorfields Eye Hospital, Addenbrooke's Hospital, University College London, MRC Mitochondrial Biology Unit, University of Bologna, Wills Eye Hospital, Fondation Ophtalmologique Adolphe de Rothschild, University of California, Los Angeles, Vita-Salute San Raffaele University, Institut Català de Retina, Centre Hospitalier Universitaire d'Angers, Massachusetts Eye and Ear Infirmary, Eye Center, GenSight Biologics (France), Centre National de la Recherche Scientifique, Institut de la Vision, Inserm, University of Pittsburgh, Centre hospitalier national d'ophtalmologie des Quinze-Vingts, Sorbonne Université
Language: English
Subfield: Genetics
Open Access status: closed
OpenAlex ID: W4389460397
FWCI: 0.141
Cites:
Cited by: 1
Related to: 10