Nicholas D Weber
Overview
Explore the profile of Nicholas D Weber including associated specialties, affiliations and a list of published articles.
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16
Citations
325
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Recent Articles
1.
Llanos-Ardaiz A, Lantero A, Neri L, Mauleon I, Ruiz de Galarreta M, Trigueros-Motos L, et al.
Int J Mol Sci
. 2024 Dec;
25(23).
PMID: 39684442
Adeno-associated viral (AAV) vector-mediated gene therapy has emerged as a promising alternative to liver transplantation for monogenic metabolic hepatic diseases. AAVs are non-integrative vectors that are maintained primarily as episomes...
2.
Weber N, Odriozola L, Ros-Ganan I, Garcia-Porrero G, Salas D, Argemi J, et al.
JHEP Rep
. 2023 Apr;
5(5):100713.
PMID: 37096142
Background & Aims: Gene therapy using recombinant adeno-associated virus (rAAV) vector carrying multidrug resistance protein 3 (MDR3) coding sequence (AAV8-MDR3) represents a potential curative treatment for progressive familial intrahepatic cholestasis...
3.
Zabaleta N, Unzu C, Weber N, Gonzalez-Aseguinolaza G
Nat Rev Gastroenterol Hepatol
. 2023 Jan;
20(5):288-305.
PMID: 36646909
Gene therapy is poised to revolutionize modern medicine, with seemingly unlimited potential for treating and curing genetic disorders. For otherwise incurable indications, including most inherited metabolic liver disorders, gene therapy...
4.
Martinez-Garcia J, Molina A, Gonzalez-Aseguinolaza G, Weber N, Smerdou C
Biomedicines
. 2022 Jun;
10(6).
PMID: 35740260
Cholestatic diseases can be caused by the dysfunction of transporters involved in hepatobiliary circulation. Although pharmacological treatments constitute the current standard of care for these diseases, none are curative, with...
5.
Martinez-Garcia J, Molina M, Odriozola L, Molina A, Gonzalez-Aseguinolaza G, Weber N, et al.
Cell Biosci
. 2022 Jun;
12(1):79.
PMID: 35641984
Background: Bile acid (BA) homeostasis is mainly regulated by bile salt excretory pump (BSEP), a hepatocyte transporter that transfers BAs to the bile. BSEP expression is regulated by BA levels...
6.
Zabaleta N, Torella L, Weber N, Gonzalez-Aseguinolaza G
Hepatology
. 2022 Mar;
76(3):869-887.
PMID: 35243655
The efficient delivery of RNA molecules to restore the expression of a missing or inadequately functioning protein in a target cell and the intentional specific modification of the host genome...
7.
Weber N, Martinez-Garcia J, Gonzalez-Aseguinolaza G
J Hepatol
. 2021 Oct;
76(3):749-751.
PMID: 34626730
No abstract available.
8.
Maestro S, Weber N, Zabaleta N, Aldabe R, Gonzalez-Aseguinolaza G
JHEP Rep
. 2021 Jun;
3(4):100300.
PMID: 34159305
Gene therapy is becoming an increasingly valuable tool to treat many genetic diseases with no or limited treatment options. This is the case for hundreds of monogenic metabolic disorders of...
9.
Kaiser R, Weber N, Trigueros-Motos L, Allen K, Martinez M, Cao W, et al.
J Inherit Metab Dis
. 2021 Apr;
44(6):1369-1381.
PMID: 33896013
Phenylketonuria (PKU) is the most common inborn error of metabolism of the liver, and results from mutations of both alleles of the phenylalanine hydroxylase gene (PAH). As such, it is...
10.
Stone D, Long K, Loprieno M, De Silva Feelixge H, Kenkel E, Liley R, et al.
Mol Ther Methods Clin Dev
. 2021 Jan;
20:258-275.
PMID: 33473359
Chronic hepatitis B virus (HBV) infection is a major public health problem. New treatment approaches are needed because current treatments do not target covalently closed circular DNA (cccDNA), the template...