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Liver-directed gene therapy results in long-term correction of progressive familial intrahepatic cholestasis type 3 in mice. [electronic resource] by
- Aronson, Sem J
- Bakker, Robert S
- Shi, Xiaoxia
- Duijst, Suzanne
- Ten Bloemendaal, Lysbeth
- de Waart, Dirk R
- Verheij, Joanne
- Ronzitti, Giuseppe
- Oude Elferink, Ronald P
- Beuers, Ulrich
- Paulusma, Coen C
- Bosma, Piter J
Producer: 20201214
In:
Journal of hepatology vol. 71
Availability: No items available.
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Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome. [electronic resource] by
- Aronson, Sem J
- Veron, Philippe
- Collaud, Fanny
- Hubert, Aurélie
- Delahais, Virginie
- Honnet, Géraldine
- de Knegt, Robert J
- Junge, Norman
- Baumann, Ulrich
- Di Giorgio, Angelo
- D'Antiga, Lorenzo
- Ginocchio, Virginia M
- Brunetti-Pierri, Nicola
- Labrune, Philippe
- Beuers, Ulrich
- Bosma, Piter J
- Mingozzi, Federico
Producer: 20200327
In:
Human gene therapy vol. 30
Availability: No items available.
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Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome. [electronic resource] by
- Collaud, Fanny
- Bortolussi, Giulia
- Guianvarc'h, Laurence
- Aronson, Sem J
- Bordet, Thierry
- Veron, Philippe
- Charles, Severine
- Vidal, Patrice
- Sola, Marcelo Simon
- Rundwasser, Stephanie
- Dufour, Delphine G
- Lacoste, Florence
- Luc, Cyril
- Wittenberghe, Laetitia V
- Martin, Samia
- Le Bec, Christine
- Bosma, Piter J
- Muro, Andres F
- Ronzitti, Giuseppe
- Hebben, Matthias
- Mingozzi, Federico
Publication details: Molecular therapy. Methods & clinical development Mar 2019
In:
Molecular therapy. Methods & clinical development vol. 12
Availability: No items available.
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