Mauro Biffi

The San Raffaele Telethon Institute for Gene Therapy — Italy

Biochemistry, Genetics and Molecular Biology · Life Sciences

16h-index2.6kcitations35works5.62yr avg
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Research Topics

Virus-based gene therapy research(12), CRISPR and Genetic Engineering(9), Pluripotent Stem Cells Research(5), RNA Interference and Gene Delivery(5), Viral gastroenteritis research and epidemiology(5)

Publications35 total

Tweaking lentiviral vector design balances efficacy and safety in liver-directed gene therapy for familial hypercholesterolemia
Molecular Therapy·2025
Spatiotemporal liver dynamics shape hepatocellular heterogeneity and impact in vivo gene engineering
Journal of Hepatology·2025· 3 citedOpen Access
In vivo haemopoietic stem cell gene therapy enabled by postnatal trafficking
Nature·2025· 9 citedOpen Access
Enhancing the potency of in vivo lentiviral vector mediated gene therapy to hepatocytes
Nature Communications·2025· 9 citedOpen Access
Liver fibrosis negatively impacts in vivo gene transfer to murine hepatocytes
Nature Communications·2025· 6 citedOpen Access
Identification of hepatocyte-primed cholangiocytes in the homeostatic liver by in vivo lentiviral gene transfer to mice and non-human primates
Cell Reports·2025· 3 citedOpen Access
GP64-pseudotyped lentiviral vectors target liver endothelial cells and correct hemophilia A mice
EMBO Molecular Medicine·2024· 14 citedOpen Access
Liver fibrosis negatively impacts in vivo gene transfer to hepatocytes
2024· 1 citedOpen Access
Spatio-temporal dynamics of the liver shapes hepatocytes heterogeneity and impacts in vivo gene transfer and editing
Research Square (Research Square)·2023· 1 citedOpen Access
Identification of Hepatocyte-Primed Biliary Epithelial Cells in the Homeostatic Liver by <i>in vivo</i> Lentiviral Gene Transfer to Mice and Non-Human Primates
SSRN Electronic Journal·2023Open Access
Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates
Nature Communications·2022· 41 citedOpen Access
Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates
Research Square (Research Square)·2021· 3 citedOpen Access
Laboratory-Scale Lentiviral Vector Production and Purification for Enhanced Ex Vivo and In Vivo Genetic Engineering
Molecular Therapy — Methods & Clinical Development·2020· 36 citedOpen Access
Phagocytosis-shielded lentiviral vectors improve liver gene therapy in nonhuman primates
Science Translational Medicine·2019· 126 citedOpen Access
Liver-Directed Gene Therapy for Hemophilia B with Immune Stealth Lentiviral Vectors
Blood·2017· 5 cited
Genome editing for scalable production of alloantigen‐free lentiviral vectors for <i>in vivo</i> gene therapy
EMBO Molecular Medicine·2017· 59 citedOpen Access
Inheritable Silencing of Endogenous Genes by Hit-and-Run Targeted Epigenetic Editing
Cell·2016· 500 citedOpen Access
729. Inheritable Silencing of Endogenous Gene by Hit-and-Run Targeted Epigenetic Editing
Molecular Therapy·2016· 2 citedOpen Access
690. Permanent Epigenetic Silencing of Human Genes With Artificial Transcriptional Repressors
Molecular Therapy·2015Open Access
Calcitriol derivatives with two different side chains at C-20
The Journal of Steroid Biochemistry and Molecular Biology·2007· 20 cited
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Frequent Co-authors

Alessio Cantore(16), Luigi Naldini(13), Michela Milani(13), Fabio Russo(13), Francesco Starinieri(9), Francesco Sinigaglia(8), Anna Fabiano(8), Lars Rogge(7), Andrea Annoni(7), Francesca Sanvito(7), Angelo Lombardo(6), Eugenio Montini(6), Rossana Norata(6), David H. Presky(5), Chiara Brombin(5), Cesare Canepari(5), Chiara Simoni(5), Monica Volpin(5), Stefano Beretta(5), Nadia Passini(4)