Elena Barbon

IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies

Christian Leborgne, Elena Barbon, J M Alexander et al. · Nature Medicine · 2020 · 295 citations · Full text

Vivo Gene Therapy, Autoimmune Disease, Igg-cleaving Endopeptidase Enables +8

Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction

Zachary Fitzpatrick, Christian Leborgne, Elena Barbon et al. · Molecular Therapy — Methods & Clinical Development · 2018 · 193 citations · Full text

Exon-specific U1 snRNAs improve <i>ELP1</i> exon 20 definition and rescue ELP1 protein expression in a familial dysautonomia mouse model

Irving Donadon, Mirko Pinotti, Katarzyna Rajkowska et al. · Human Molecular Genetics · 2018 · 54 citations · Full text

Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System

Annalisa Lattanzi, Stéphanie Duguez, Arianna Moiani et al. · Molecular Therapy — Nucleic Acids · 2017 · 53 citations · Full text

Molecular Basis and Therapeutic Strategies to Rescue Factor IX Variants That Affect Splicing and Protein Function

Mojca Tajnik, Malgorzata Ewa Rogalska, Erica Bussani et al. · PLoS Genetics · 2016 · 41 citations · Full text

Genetics, Rna Splicing, Genomic Mechanism +16