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

P Wegen, Rogier Louwen, Ali Imam, Luna Offerman, M Sinaasappel, Frank Grosveld, Bob Scholte

Research output: Contribution to journalArticleAcademicpeer-review

31 Citations (Scopus)
Original languageUndefined/Unknown
Pages (from-to)374-381
Number of pages8
JournalMolecular Therapy
Volume13
Issue number2
DOIs
Publication statusPublished - 2006

Research programs

  • EMC MM-04-54-07

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