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Title
Targeted gene therapies: tools, applications, optimization
Authors
Keywords
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Journal
CRITICAL REVIEWS IN BIOCHEMISTRY AND MOLECULAR BIOLOGY
Volume 47, Issue 3, Pages 264-281
Publisher
Informa UK Limited
Online
2012-04-25
DOI
10.3109/10409238.2012.658112
References
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- (2011) J. R. LaRocque et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
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