Author

Samantha Scaramuzza

The San Raffaele Telethon Institute for Gene TherapyORCID 0000-0003-2903-3650Unclaimed ORCID profile · follow to get notified of new preprints

Biology Disciplines

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Signature Assays, Organisms & Methods

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Approach:experimental assay· 2 Organism:human· 2

Frequent Co-Authors

Annarita MiccioiD· 2 shared Cécile MassoniD· 2 shared Giuliana FerrariiD· 2 shared Mario AmendolaiD· 2 shared Oriana RomanoiD· 2 shared Pierre MartinucciiD· 2 shared Alexandra TachtsidiiD· 1 shared Anne ChalumeauiD· 1 shared

Activity by Year

2022-2025 · peak 1/yr

Co-Author Collaboration Network

10 top collaborators · 42 mutual co-author links

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2 shared papersGenetics

Annarita MiccioORCID

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2 papers

Recent·Most cited

GeneticsbioRxivPreprintAug 21, 2025

Base editing of β0 thalassemia mutations as a therapeutic strategy for β-hemoglobinopathies: efficacy and genotoxicity studies

Giulia Hardouin, Pierre Martinucci, Samantha Scaramuzza…·Annarita Miccio

Preclinical safety and efficacy studies of a new gene therapy approach for patients with severe β-hemoglobinopathies.

DOI 10.1101/2025.08.21.671453

humanexperimental assay

𝕏 PostBluesky

GeneticsbioRxivPreprintDec 31, 2022

Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies

Mégane Brusson, Anne Chalumeau, Pierre Martinucci…·Annarita Miccio

This study develops a bifunctional lentiviral vector for sickle cell disease that combines gene addition and gene silencing to reduce HbS levels and improve treatment efficacy.

2 citations

humanexperimental assay

𝕏 PostBluesky