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, Severine Charles, Christian Leborgne, Patrice Vidal, Samia Martin, Bernard Gjata, Marcelo Simon Sola, Laetitia van Wittenberghe, Alban Vignaud, Philippe Veron, Piter J Bosma, Andres F Muro, Federico Mingozzi

Open source

DOI
10.1038/mtm.2016.49
Published
2016
Container
Molecular Therapy - Methods & Clinical Development
Publisher
Elsevier BV
Open access
unknown

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BibTeX

@article{allodium:10.1038/mtm.2016.49,
  title = {A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome},
  author = {Giuseppe Ronzitti and Giulia Bortolussi and Remco van Dijk and Fanny Collaud and Severine Charles and Christian Leborgne and Patrice Vidal and Samia Martin and Bernard Gjata and Marcelo Simon Sola and Laetitia van Wittenberghe and Alban Vignaud and Philippe Veron and Piter J Bosma and Andres F Muro and Federico Mingozzi},
  year = {2016},
  journal = {Molecular Therapy - Methods \& Clinical Development},
  doi = {10.1038/mtm.2016.49},
  url = {https://doi.org/10.1038/mtm.2016.49}
}

RIS

TY  - JOUR
TI  - A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome
AU  - Giuseppe Ronzitti
AU  - Giulia Bortolussi
AU  - Remco van Dijk
AU  - Fanny Collaud
AU  - Severine Charles
AU  - Christian Leborgne
AU  - Patrice Vidal
AU  - Samia Martin
AU  - Bernard Gjata
AU  - Marcelo Simon Sola
AU  - Laetitia van Wittenberghe
AU  - Alban Vignaud
AU  - Philippe Veron
AU  - Piter J Bosma
AU  - Andres F Muro
AU  - Federico Mingozzi
PY  - 2016
JO  - Molecular Therapy - Methods & Clinical Development
DO  - 10.1038/mtm.2016.49
UR  - https://doi.org/10.1038/mtm.2016.49
ER  - 

APA

Ronzitti, G., Bortolussi, G., Dijk, R. V., Collaud, F., Charles, S., Leborgne, C., Vidal, P., Martin, S., Gjata, B., Sola, M. S., Wittenberghe, L. V., Vignaud, A., Veron, P., Bosma, P. J., Muro, A. F., & Mingozzi, F. (2016). A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome. Molecular Therapy - Methods & Clinical Development. https://doi.org/10.1038/mtm.2016.49

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