Weber ND - Search Results

1

Gene Therapy for Acquired and Genetic Cholestasis.

Martínez-García J, Molina A, González-Aseguinolaza G, Weber ND, Smerdou C. Martínez-García J, et al. Among authors: weber nd. Biomedicines. 2022 May 26;10(6):1238. doi: 10.3390/biomedicines10061238. Biomedicines. 2022. PMID: 35740260 Free PMC article. Review.

2

Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model.

Weber ND, Odriozola L, Martínez-García J, Ferrer V, Douar A, Bénichou B, González-Aseguinolaza G, Smerdou C. Weber ND, et al. Nat Commun. 2019 Dec 13;10(1):5694. doi: 10.1038/s41467-019-13614-3. Nat Commun. 2019. PMID: 31836711 Free PMC article.

3

Use of an adeno-associated virus serotype Anc80 to provide durable cure of phenylketonuria in a mouse model.

Kaiser RA, Weber ND, Trigueros-Motos L, Allen KL, Martinez M, Cao W, VanLith CJ, Hillin LG, Douar A, González-Aseguinolaza G, Aldabe R, Lillegard JB. Kaiser RA, et al. Among authors: weber nd. J Inherit Metab Dis. 2021 Nov;44(6):1369-1381. doi: 10.1002/jimd.12392. Epub 2021 Aug 19. J Inherit Metab Dis. 2021. PMID: 33896013 Free PMC article.

4

Novel vectors and approaches for gene therapy in liver diseases.

Maestro S, Weber ND, Zabaleta N, Aldabe R, Gonzalez-Aseguinolaza G. Maestro S, et al. Among authors: weber nd. JHEP Rep. 2021 Apr 30;3(4):100300. doi: 10.1016/j.jhepr.2021.100300. eCollection 2021 Aug. JHEP Rep. 2021. PMID: 34159305 Free PMC article. Review.

5

Comment on "Synthetic human ABCB4 mRNA therapy rescues severe liver disease phenotype in a BALB/c.Abcb4^-/- mouse model of PFIC3".

Weber ND, Martínez-García J, González-Aseguinolaza G. Weber ND, et al. J Hepatol. 2022 Mar;76(3):749-751. doi: 10.1016/j.jhep.2021.09.033. Epub 2021 Oct 6. J Hepatol. 2022. PMID: 34626730 No abstract available.

6

mRNA and gene editing: Late breaking therapies in liver diseases.

Zabaleta N, Torella L, Weber ND, Gonzalez-Aseguinolaza G. Zabaleta N, et al. Among authors: weber nd. Hepatology. 2022 Sep;76(3):869-887. doi: 10.1002/hep.32441. Epub 2022 Apr 1. Hepatology. 2022. PMID: 35243655 Free PMC article. Review.

7

A minimal bile salt excretory pump promoter allows bile acid-driven physiological regulation of transgene expression from a gene therapy vector.

Martínez-García J, Molina M, Odriozola L, Molina A, González-Aseguinolaza G, Weber ND, Smerdou C. Martínez-García J, et al. Among authors: weber nd. Cell Biosci. 2022 May 31;12(1):79. doi: 10.1186/s13578-022-00803-9. Cell Biosci. 2022. PMID: 35641984 Free PMC article.

8

Gene therapy for liver diseases - progress and challenges.

Zabaleta N, Unzu C, Weber ND, Gonzalez-Aseguinolaza G. Zabaleta N, et al. Among authors: weber nd. Nat Rev Gastroenterol Hepatol. 2023 May;20(5):288-305. doi: 10.1038/s41575-022-00729-0. Epub 2023 Jan 16. Nat Rev Gastroenterol Hepatol. 2023. PMID: 36646909 Review.

9

Rescue of infant progressive familial intrahepatic cholestasis type 3 mice by repeated dosing of AAV gene therapy.

Weber ND, Odriozola L, Ros-Gañán I, García-Porrero G, Salas D, Argemi J, Combal JP, Kishimoto TK, González-Aseguinolaza G. Weber ND, et al. JHEP Rep. 2023 Feb 24;5(5):100713. doi: 10.1016/j.jhepr.2023.100713. eCollection 2023 May. JHEP Rep. 2023. PMID: 37096142 Free PMC article.

10

In Vivo Selection of S/MAR Sequences to Favour AAV Episomal Maintenance in Dividing Cells.

Llanos-Ardaiz A, Lantero A, Neri L, Mauleón I, Ruiz de Galarreta M, Trigueros-Motos L, Weber ND, Ferrer V, Aldabe R, Gonzalez-Aseguinolaza G. Llanos-Ardaiz A, et al. Among authors: weber nd. Int J Mol Sci. 2024 Nov 27;25(23):12734. doi: 10.3390/ijms252312734. Int J Mol Sci. 2024. PMID: 39684442 Free PMC article.

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