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In Vivo Gene Transfer Strategies to Achieve Partial Correction of von Willebrand Disease

Authors
Journal
Human Gene Therapy
1043-0342
Publisher
Mary Ann Liebert
Publication Date
Volume
23
Issue
6
Identifiers
DOI: 10.1089/hum.2011.238
Keywords
  • Research Articles

Abstract

Wang and colleagues evaluate the ability of a lentiviral vector to transfer intact murine von Willebrand factor (VWF) cDNA into the neonatal liver of VWF−/− mice. The lentivirus generates VWF multimers and partially corrects the coagulation defect on a persistent basis in 33% of treated mice.

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