Alessandra Iaconcig
Trieste, Italy
Last Updated: 09/26/2026
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Found 14 publications
Liver Gene Therapy in Fabry Disease Mice With Low Doses of rAAV2/8 Expressing a Codon-Optimized hGLA cDNA Results in Long-Term Disease Correction.
Journal: Journal of inherited metabolic disease
Published: November 24, 2025
Rapamycin nanoparticles mitigate anti-AAV antibody formation in a mouse model of ornithine transcarbamylase deficiency.
Journal: Molecular therapy. Advances
Published: November 18, 2025
Lethality rescue and long-term amelioration of a citrullinemia type I mouse model by neonatal gene-targeting combined to SaCRISPR-Cas9.
Journal: Molecular therapy. Methods & clinical development
Published: May 08, 2023
CRISPR-Cas9-mediated somatic correction of a one-base deletion in the Ugt1a gene ameliorates hyperbilirubinemia in Crigler-Najjar syndrome mice.
Journal: Molecular therapy. Methods & clinical development
Published: February 10, 2023
Formation of an invasion-permissive matrix requires TGFβ/SNAIL1-regulated alternative splicing of fibronectin.
Journal: Breast cancer research : BCR
Published: November 08, 2022
Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector.
Journal: Molecular therapy. Methods & clinical development
Published: July 09, 2020
Attenuation of neuro-inflammation improves survival and neurodegeneration in a mouse model of severe neonatal hyperbilirubinemia.
Journal: Brain, behavior, and immunity
Published: December 21, 2017
Promoterless gene targeting without nucleases rescues lethality of a Crigler-Najjar syndrome mouse model.
Journal: EMBO molecular medicine
Published: July 29, 2017
Repeated AAV-mediated gene transfer by serotype switching enables long-lasting therapeutic levels of hUgt1a1 enzyme in a mouse model of Crigler-Najjar Syndrome Type I.
Journal: Gene therapy
Published: February 20, 2017
Modulation of bilirubin neurotoxicity by the Abcb1 transporter in the Ugt1-/- lethal mouse model of neonatal hyperbilirubinemia.
Journal: Human molecular genetics
Published: September 13, 2016
TDP-43 regulates β-adducin (Add2) transcript stability.
Journal: RNA biology
Published: January 21, 2015
Long-distance regulation of Add2 pre-mRNA3'end processing.
Journal: RNA biology
Published: February 16, 2013
Last Updated: 09/26/2026