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January 1, 2016Molecular Therapy — Methods & Clinical DevelopmentOpen Access

A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome

Giuseppe Ronzitti Giulia Bortolussi Remco van Dijk Fanny Collaud Séverine Charles Christian Leborgne Patrice Vidal Samia Martin Bernard Gjata Marcelo Simon Sola Laetitia van Wittenberghe Alban Vignaud P. Véron Piter J. Bosma Andrés F. Muro Federico Mingozzi

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Abstract

mice. We also showed long-term (>1 year) correction of the disease in Gunn rats. These results support further translation of the approach to humans.

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Ronzitti et al. (2016) studied this question.

synapsesocial.com/papers/6a02630ca28f8b391669b66d https://doi.org/10.1038/mtm.2016.49

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Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

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