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Succesful treatment of UGT1A1 deficiency in a rat model of Crigler-Najjar disease by intravenous administration of a liver-specific lentiviral vector

  • CG (Pascal) van der Wegen
  • , Rogier Louwen
  • , Ali Imam
  • , Luna Offerman
  • , M (Maarten) Sinaasappel
  • , Frank Grosveld
  • , Bob Scholte

Research output: Contribution to journalArticleAcademicpeer-review

Original languageUndefined/Unknown
Pages (from-to)374-381
Number of pages8
JournalMolecular Therapy
Volume12
Issue number2
Publication statusPublished - 2006

Research programs

  • EMC MGC-02-13-03
  • EMC MM-04-54-07

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