Successful Treatment of UGT1A1 Deficiency in a Rat Model of Crigler–Najjar Disease by Intravenous Administration of a Liver-Specific Lentiviral Vector

Molecular Therapy - Tập 13 - Trang 374-381 - 2006
Pascal van der Wegen1, Rogier Louwen1, Ali M. Imam1, Ruvalic M. Buijs-Offerman1, Maarten Sinaasappel2, Frank Grosveld1, Bob J. Scholte1
1Department of Cell Biology and Genetics, Erasmus Medical Center, P.O. Box 1738, 3000 DR Rotterdam, The Netherlands
2Department of Pediatric Gastroenterology, Erasmus Medical Center, P.O. Box 1738, 3000 DR Rotterdam, The Netherlands

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Molecular Therapy

Tập 13

374-381

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