Gene therapy for Leber congenital amaurosis: advances and future directions
Published 2012 View Full Article
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Title
Gene therapy for Leber congenital amaurosis: advances and future directions
Authors
Keywords
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Journal
GRAEFES ARCHIVE FOR CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY
Volume 250, Issue 8, Pages 1117-1128
Publisher
Springer Nature
Online
2012-05-28
DOI
10.1007/s00417-012-2028-2
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- Nanoparticles for retinal gene therapy
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- (2009) Artur V. Cideciyan et al. HUMAN GENE THERAPY
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- (2008) A. V. Cideciyan et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
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