Nicholas D. Weber
Pamplona, NC 31008, Spain
Last Updated: 09/26/2026
Save publications for later
Sign Up
Not sure about your diagnosis?
Check Your Symptoms
Found 10 publications
In Vivo Selection of S/MAR Sequences to Favour AAV Episomal Maintenance in Dividing Cells.
Journal: International journal of molecular sciences
Published: October 21, 2024
Gene therapy for liver diseases - progress and challenges.
Journal: Nature reviews. Gastroenterology & hepatology
Published: December 09, 2022
Rescue of infant progressive familial intrahepatic cholestasis type 3 mice by repeated dosing of AAV gene therapy.
Journal: JHEP reports : innovation in hepatology
Published: August 22, 2022
mRNA and gene editing: Late breaking therapies in liver diseases.
Journal: Hepatology (Baltimore, Md.)
Published: December 01, 2021
A minimal bile salt excretory pump promoter allows bile acid-driven physiological regulation of transgene expression from a gene therapy vector.
Journal: Cell & bioscience
Published: September 28, 2021
Comment on "Synthetic human ABCB4 mRNA therapy rescues severe liver disease phenotype in a BALB/c.Abcb4-/- mouse model of PFIC3".
Journal: Journal of hepatology
Published: September 20, 2021
Novel vectors and approaches for gene therapy in liver diseases.
Journal: JHEP reports : innovation in hepatology
Published: January 21, 2021
Use of an adeno-associated virus serotype Anc80 to provide durable cure of phenylketonuria in a mouse model.
Journal: Journal of inherited metabolic disease
Published: December 28, 2020
Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model.
Journal: Nature communications
Published: December 21, 2018
Showing 1-10 of 10
Last Updated: 09/26/2026