Durable and efficient gene silencing in vivo by hit-and-run epigenome editing
2024 · Nature · 156 citations
https://doi.org/10.1038/s41586-024-07087-8
S
Simone Merlin
Università degli Studi del Piemonte Orientale “Amedeo Avogadro”(IT)
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Simone Merlin is an academic researcher from Università degli Studi del Piemonte Orientale “Amedeo Avogadro”. The author has contributed to research in topics: Hemophilia Treatment and Research & Platelet Disorders and Treatments & Liver physiology and pathology. The author has an h-index of 20, co-authored 48 publications.
ORCID: 0000-0001-5209-3921Papers by this author
worksTherapeutic correction of hemophilia A by transplantation of hPSC-derived liver sinusoidal endothelial cell progenitors
2022 · Cell Reports · 22 citations
https://doi.org/10.1016/j.celrep.2022.110621
Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device
2021 · Molecular Therapy — Methods & Clinical Development · 19 citations
https://doi.org/10.1016/j.omtm.2021.10.015
Factor VIII as a potential player in cancer pathophysiology
2021 · Journal of Thrombosis and Haemostasis · 16 citations
https://doi.org/10.1111/jth.15611
Liver gene therapy with intein‐mediated F8 <i>trans</i> ‐splicing corrects mouse haemophilia A
2022 · EMBO Molecular Medicine · 13 citations
https://doi.org/10.15252/emmm.202115199
Immune tolerance promotion by LSEC-specific lentiviral vector-mediated expression of the transgene regulated by the stabilin-2 promoter
2024 · Molecular Therapy — Nucleic Acids · 7 citations
https://doi.org/10.1016/j.omtn.2024.102116
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