| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 3295776 | Gastroenterology | 2010 | 11 Pages |
Abstract
Lentiviral vectors that express UGT1A1 reduce hyperbilirubinemia in immunocompetent Gunn rats for at least 6 months. The immune response against virally expressed UGT1A1 can be circumvented by inclusion of miR-142 target sequences, which reduce vector expression in antigen-presenting cells. This lentiviral-based gene therapy approach might be developed to treat patients with CN-I.
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Authors
Françoise Schmitt, Séverine Remy, Anne Dariel, Maude Flageul, Virginie Pichard, Sébastien Boni, Claire Usal, Anne Myara, Sophie Laplanche, Ignacio Anegon, Philippe Labrune, Guillaume Podevin, Nicolas Ferry, Tuan Huy Nguyen,