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  1. 2022

  2. Disease burden and management of Crigler-Najjar syndrome: Report of a world registry

    CureCN Consortium, 1 Jul 2022, In: Liver international. 42, 7, p. 1593-1604 12 p.

    Research output: Contribution to journal › Article › Academic › peer-review

  3. What’s next in gene therapy for Crigler-Najjar syndrome?

    Aronson, S. J., Ronzitti, G. & Bosma, P. J., 2022, (E-pub ahead of print) In: Expert opinion on biological therapy.

    Research output: Contribution to journal › Editorial › Academic › peer-review

  4. 2021

  5. Long-term effects of biliverdin reductase a deficiency in ugt1−/− mice: impact on redox status and metabolism

    Bortolussi, G., Shi, X., Bloemendaal, L. T., Banerjee, B., de Waart, D. R., Baj, G., Chen, W., Oude Elferink, R. P., Beuers, U., Paulusma, C. C., Stocker, R., Muro, A. F. & Bosma, P. J., 1 Dec 2021, In: Antioxidants. 10, 12, 2029.

    Research output: Contribution to journal › Article › Academic › peer-review

  6. Reduced spontaneous itch in mouse models of cholestasis

    Langedijk, J., Bolier, R., Tolenaars, D., ten Bloemendaal, L., Duijst, S., de Waart, D., Beuers, U., Bosma, P. & Elferink, R. O., 1 Dec 2021, In: Scientific reports. 11, 1, 6127.

    Research output: Contribution to journal › Article › Academic › peer-review

  7. Therapeutic base editing in the adult liver

    Paulusma, C. C. & Bosma, P. J., Sept 2021, In: Nature Reviews Gastroenterology and Hepatology. 18, 9, p. 597-598 2 p.

    Research output: Contribution to journal › Article › Academic › peer-review

  8. Low efficacy of recombinant SV40 in Ugt1a1-/- mice with severe inherited hyperbilirubinemia

    Shi, X., Bortolussi, G., ten Bloemendaal, L., Duijst, S., Muro, A. F. & Bosma, P. J., 1 Apr 2021, In: PLoS ONE. 16, 4 April, e0250605.

    Research output: Contribution to journal › Article › Academic › peer-review

  9. 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

  10. Gene therapy for progressive familial intrahepatic cholestasis: Current progress and future prospects

    Bosma, P. J., Wits, M. & Oude-Elferink, R. P. J., 1 Jan 2021, In: International journal of molecular sciences. 22, 1, p. 1-13 13 p., 273.

    Research output: Contribution to journal › Review article › Academic › peer-review

  11. 2020

  12. An exon-specific small nuclear u1 rna (Exspeu1) improves hepatic otc expression in a splicing-defective spf /ash mouse model of ornithine transcarbamylase deficiency

    Balestra, D., Ferrarese, M., Lombardi, S., Ziliotto, N., Branchini, A., Petersen, N., Bosma, P., Pinotti, M. & van de Graaf, S. F. J., 2 Nov 2020, In: International journal of molecular sciences. 21, 22, p. 1-9 9 p., 8735.

    Research output: Contribution to journal › Article › Academic › peer-review

  13. 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

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