Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation

Search Page

Filters

My NCBI Filters

Text availability

Article attribute

Article type

Publication date

Search Results

81 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Results By Year timeline is not available.
Page 1
Gene Therapy in Patients with the Crigler-Najjar Syndrome.
D'Antiga L, Beuers U, Ronzitti G, Brunetti-Pierri N, Baumann U, Di Giorgio A, Aronson S, Hubert A, Romano R, Junge N, Bosma P, Bortolussi G, Muro AF, Soumoudronga RF, Veron P, Collaud F, Knuchel-Legendre N, Labrune P, Mingozzi F. D'Antiga L, et al. Among authors: muro af. N Engl J Med. 2023 Aug 17;389(7):620-631. doi: 10.1056/NEJMoa2214084. N Engl J Med. 2023. PMID: 37585628 Clinical Trial.
A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome.
Ronzitti G, Bortolussi G, van Dijk R, Collaud F, Charles S, Leborgne C, Vidal P, Martin S, Gjata B, Sola MS, van Wittenberghe L, Vignaud A, Veron P, Bosma PJ, Muro AF, Mingozzi F. Ronzitti G, et al. Among authors: muro af. Mol Ther Methods Clin Dev. 2016 Jul 20;3:16049. doi: 10.1038/mtm.2016.49. eCollection 2016. Mol Ther Methods Clin Dev. 2016. PMID: 27722180 Free PMC article.
Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome.
Collaud F, Bortolussi G, Guianvarc'h L, Aronson SJ, Bordet T, Veron P, Charles S, Vidal P, Sola MS, Rundwasser S, Dufour DG, Lacoste F, Luc C, Wittenberghe LV, Martin S, Le Bec C, Bosma PJ, Muro AF, Ronzitti G, Hebben M, Mingozzi F. Collaud F, et al. Among authors: muro af. Mol Ther Methods Clin Dev. 2018 Dec 31;12:157-174. doi: 10.1016/j.omtm.2018.12.011. eCollection 2019 Mar 15. Mol Ther Methods Clin Dev. 2018. PMID: 30705921 Free PMC article.
A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene.
Aronson SJ, Bakker RS, Moenis S, van Dijk R, Bortolussi G, Collaud F, Shi X, Duijst S, Ten Bloemendaal L, Ronzitti G, Muro AF, Mingozzi F, Beuers U, Bosma PJ. Aronson SJ, et al. Among authors: muro af. Mol Ther Methods Clin Dev. 2020 Jun 3;18:250-258. doi: 10.1016/j.omtm.2020.06.002. eCollection 2020 Sep 11. Mol Ther Methods Clin Dev. 2020. PMID: 32637454 Free PMC article.
Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients.
Shi X, Aronson SJ, Ten Bloemendaal L, Duijst S, Bakker RS, de Waart DR, Bortolussi G, Collaud F, Oude Elferink RP, Muro AF, Mingozzi F, Ronzitti G, Bosma PJ. Shi X, et al. Among authors: muro af. Mol Ther Methods Clin Dev. 2020 Dec 3;20:287-297. doi: 10.1016/j.omtm.2020.11.016. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2020. PMID: 33511243 Free PMC article.
Experimental models assessing bilirubin neurotoxicity.
Bortolussi G, Muro AF. Bortolussi G, et al. Among authors: muro af. Pediatr Res. 2020 Jan;87(1):17-25. doi: 10.1038/s41390-019-0570-x. Epub 2019 Sep 7. Pediatr Res. 2020. PMID: 31493769 Review.
Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector.
De Sabbata G, Boisgerault F, Guarnaccia C, Iaconcig A, Bortolussi G, Collaud F, Ronzitti G, Sola MS, Vidal P, Rouillon J, Charles S, Nicastro E, D'Antiga L, Ilyinskii P, Mingozzi F, Kishimoto TK, Muro AF. De Sabbata G, et al. Among authors: muro af. Mol Ther Methods Clin Dev. 2020 Nov 17;20:169-180. doi: 10.1016/j.omtm.2020.11.005. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2020. PMID: 33473356 Free PMC article.
81 results