Federico Mingozzi

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

Amit Nathwani, Ulreke M. Reiss, Edward G. D. Tuddenham et al. · New England Journal of Medicine · 2014 · 1.3K citations · Full text

Medicine, Genetics, Molecular Genetics +17

Humoral immunity to AAV vectors in gene therapy: challenges and potential solutions.

Elisa Masat, Giulia Pavani, Federico Mingozzi · PubMed · 2013 · 89 citations

Life Sciences, Biotechnology, Immunology +15

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 · 57 citations · Full text

Life Sciences, Genetics, Molecular Biology +16

Autophagy determines efficiency of liver‐directed gene therapy with adeno‐associated viral vectors

Marianna Hösel, Anke Huber, Susanne Bohlen et al. · Hepatology · 2017 · 46 citations · Full text

Life Sciences, Cell Biology, Molecular Medicine +17

Enhanced Efficacy from Gene Therapy in Pompe Disease Using Coreceptor Blockade

Sang-oh Han, Songtao Li, Elizabeth D. Brooks et al. · Human Gene Therapy · 2014 · 30 citations · Full text

Life Sciences, Immunology, Molecular Medicine +14