-
Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients
Shi, X., Aronson, S. J., ten Bloemendaal, L., Duijst, S., Bakker, R. S., de Waart, D. R., Bortolussi, G., Collaud, F., Oude Elferink, R. P., Muro, A. F., Mingozzi, F., Ronzitti, G. & Bosma, P. J., 12 Mar 2021, In: Molecular therapy. Methods & clinical development. 20, p. 287-297 11 p.Research output: Contribution to journal › Article* › Academic › peer-review
-
A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene
Aronson, S. J., Bakker, R. S., Moenis, S., van Dijk, R., Bortolussi, G., Collaud, F., Shi, X., Duijst, S., ten Bloemendaal, L., Ronzitti, G., Muro, A. F., Mingozzi, F., Beuers, U. & Bosma, P. J., 11 Sept 2020, In: Molecular therapy. Methods & clinical development. 18, p. 250-258 9 p.Research output: Contribution to journal › Article* › Academic › peer-review
-
Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome
Aronson, S. J., Veron, P., Collaud, F., Hubert, A., Delahais, V., Honnet, G. R., de Knegt, R. J., Junge, N., Baumann, U., di Giorgio, A., D'Antiga, L., Ginocchio, V. M., Brunetti-Pierri, N., Labrune, P., Beuers, U., Bosma, P. J. & Mingozzi, F., Oct 2019, In: Human gene therapy. 30, 10, p. 1297-1305 9 p.Research output: Contribution to journal › Article* › Academic › peer-review
- All publications
