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Methods in Cell Biology|February 22, 2026
Adeno-associated viral vector administration to the inner ear and phenotype evaluation in a mouse model of hearing lossSergio Isola, Carmen Unzu
Nature Reviews. Gastroenterology & Hepatology|January 16, 2023
Gene therapy for liver diseases - progress and challengesNerea Zabaleta, Carmen Unzu, Nicholas D Weber, et al.
Stem Cells International|February 17, 2016
Human Hepatocyte-Derived Induced Pluripotent Stem Cells: MYC Expression, Similarities to Human Germ Cell Tumors, and Safety IssuesCarmen Unzu, Marc Friedli, Alexis Bosman, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|April 23, 2025
Clinical perspective: Advancing hemophilia treatment through gene therapy approachesCourtney D Thornburg, Steve W Pipe, Alessio Cantore, et al.
Human Gene Therapy|September 6, 2023
Understanding and Tackling Immune Responses to Adeno-Associated Viral VectorsHelena Costa-Verdera, Carmen Unzu, Erika Valeri, et al.
Human Molecular Genetics|April 9, 2013
Helper-dependent adenoviral liver gene therapy protects against induced attacks and corrects protein folding stress in acute intermittent porphyria miceCarmen Unzu, Ana Sampedro, Itsaso Mauleón, et al.
Plos One|March 14, 2012
Renal failure affects the enzymatic activities of the three first steps in hepatic heme biosynthesis in the acute intermittent porphyria mouseCarmen Unzu, Ana Sampedro, Eliane Sardh, et al.
Pharmaceutics|October 28, 2023
Topoisomerase Inhibitors Increase Episomal DNA Expression by Inducing the Integration of Episomal DNA in Hepatic CellsAndoni Gómez-Moreno, Enara San Sebastian, Jennifer Moya, et al.
Molecular Therapy. Methods & Clinical Development|January 19, 2023
High-efficiency purification of divergent AAV serotypes using AAVX affinity chromatographyMichael Florea, Fotini Nicolaou, Simon Pacouret, et al.
Journal of Hepatology|October 10, 2009
Porphobilinogen deaminase over-expression in hepatocytes, but not in erythrocytes, prevents accumulation of toxic porphyrin precursors in a mouse model of acute intermittent porphyriaCarmen Unzu, Ana Sampedro, Itsaso Mauleón, et al.
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