Brunetti-Pierri N - Search Results

1

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

Aronson SJ, Junge N, Trabelsi M, Kelmemi W, Hubert A, Brigatti KW, Fox MD, de Knegt RJ, Escher JC, Ginocchio VM, Iorio R, Zhu Y, Özçay F, Rahim F, El-Shabrawi MHF, Shteyer E, Di Giorgio A, D'Antiga L, Mingozzi F, Brunetti-Pierri N, Strauss KA, Labrune P, Mrad R, Baumann U, Beuers U, Bosma PJ; CureCN Consortium. Aronson SJ, et al. Liver Int. 2022 Jul;42(7):1593-1604. doi: 10.1111/liv.15239. Epub 2022 Mar 29. Liver Int. 2022. PMID: 35274801 No abstract available.

2

Progress toward improved therapies for inborn errors of metabolism.

Ginocchio VM, Brunetti-Pierri N. Ginocchio VM, et al. Hum Mol Genet. 2016 Apr 15;25(R1):R27-35. doi: 10.1093/hmg/ddv418. Epub 2015 Oct 6. Hum Mol Genet. 2016. PMID: 26443595 Review.

3

Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome.

Aronson SJ, Veron P, Collaud F, Hubert A, Delahais V, Honnet G, de Knegt RJ, Junge N, Baumann U, Di Giorgio A, D'Antiga L, Ginocchio VM, Brunetti-Pierri N, Labrune P, Beuers U, Bosma PJ, Mingozzi F. Aronson SJ, et al. Hum Gene Ther. 2019 Oct;30(10):1297-1305. doi: 10.1089/hum.2019.143. Hum Gene Ther. 2019. PMID: 31502485 Free PMC article.

4

Current Status on Clinical Development of Adeno-Associated Virus-Mediated Liver-Directed Gene Therapy for Inborn Errors of Metabolism.

Ginocchio VM, Ferla R, Auricchio A, Brunetti-Pierri N. Ginocchio VM, et al. Hum Gene Ther. 2019 Oct;30(10):1204-1210. doi: 10.1089/hum.2019.151. Epub 2019 Sep 13. Hum Gene Ther. 2019. PMID: 31517544 Review.

5

Characterization of liver involvement in defects of cholesterol biosynthesis: long-term follow-up and review.

Rossi M, Vajro P, Iorio R, Battagliese A, Brunetti-Pierri N, Corso G, Di Rocco M, Ferrari P, Rivasi F, Vecchione R, Andria G, Parenti G. Rossi M, et al. Am J Med Genet A. 2005 Jan 15;132A(2):144-51. doi: 10.1002/ajmg.a.30426. Am J Med Genet A. 2005. PMID: 15580635 Review.

6

Improved efficacy and reduced toxicity by ultrasound-guided intrahepatic injections of helper-dependent adenoviral vector in Gunn rats.

Pastore N, Nusco E, Piccolo P, Castaldo S, Vaníkova J, Vetrini F, Palmer DJ, Vitek L, Ng P, Brunetti-Pierri N. Pastore N, et al. Hum Gene Ther Methods. 2013 Oct;24(5):321-7. doi: 10.1089/hgtb.2013.108. Hum Gene Ther Methods. 2013. PMID: 23947957

7

Correction of hyperbilirubinemia in gunn rats by surgical delivery of low doses of helper-dependent adenoviral vectors.

Schmitt F, Pastore N, Abarrategui-Pontes C, Flageul M, Myara A, Laplanche S, Labrune P, Podevin G, Nguyen TH, Brunetti-Pierri N. Schmitt F, et al. Hum Gene Ther Methods. 2014 Jun;25(3):181-6. doi: 10.1089/hgtb.2013.236. Epub 2014 Apr 14. Hum Gene Ther Methods. 2014. PMID: 24593043

8

Correction of hyperbilirubinemia in gunn rats using clinically relevant low doses of helper-dependent adenoviral vectors.

Dimmock D, Brunetti-Pierri N, Palmer DJ, Beaudet AL, Ng P. Dimmock D, et al. Hum Gene Ther. 2011 Apr;22(4):483-8. doi: 10.1089/hum.2010.167. Epub 2011 Feb 16. Hum Gene Ther. 2011. PMID: 20973621 Free PMC article.

9

Sustained reduction of hyperbilirubinemia in Gunn rats after adeno-associated virus-mediated gene transfer of bilirubin UDP-glucuronosyltransferase isozyme 1A1 to skeletal muscle.

Pastore N, Nusco E, Vaníkova J, Sepe RM, Vetrini F, McDonagh A, Auricchio A, Vitek L, Brunetti-Pierri N. Pastore N, et al. Hum Gene Ther. 2012 Oct;23(10):1082-9. doi: 10.1089/hum.2012.018. Epub 2012 Aug 27. Hum Gene Ther. 2012. PMID: 22765254

10

Recurrent de novo missense variants in GNB2 can cause syndromic intellectual disability.

Tan NB, Pagnamenta AT, Ferla MP, Gadian J, Chung BH, Chan MC, Fung JL, Cook E, Guter S, Boschann F, Heinen A, Schallner J, Mignot C, Keren B, Whalen S, Sarret C, Mittag D, Demmer L, Stapleton R, Saida K, Matsumoto N, Miyake N, Sheffer R, Mor-Shaked H, Barnett CP, Byrne AB, Scott HS, Kraus A, Cappuccio G, Brunetti-Pierri N, Iorio R, Di Dato F, Pais LS, Yeung A, Tan TY, Taylor JC, Christodoulou J, White SM. Tan NB, et al. J Med Genet. 2022 May;59(5):511-516. doi: 10.1136/jmedgenet-2020-107462. Epub 2021 Jun 28. J Med Genet. 2022. PMID: 34183358

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