Mario Amendola

8PUBLICATIONS
44CO-AUTHORS
Gene and molecular therapyChemical and thermal processes in energy and combustionInfant and child healthHaematologyMedical devices
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Publications (8)

|Nov 26, 2025
Base editing of β0-thalassemia mutations as a therapeutic strategy for severe β-hemoglobinopathies.

Giulia Hardouin, Pierre Martinucci, Samantha Scaramuzza

|Aug 26, 2025
A prime editing strategy to rewrite the γ-globin promoters and reactivate fetal hemoglobin for sickle cell disease.

Anne Chalumeau, Maria Bou Dames, Letizia Fontana

|Apr 11, 2025
Rescue of lysosomal acid lipase deficiency in mice by rAAV8 liver gene transfer.

Marine Laurent, Rim Harb, Christine Jenny

|Nov 05, 2022
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression.

Panagiotis Antoniou, Giulia Hardouin, Pierre Martinucci

|Sep 12, 2020
Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype.

Leslie Weber, Giacomo Frati, Tristan Felix

|Aug 15, 2020
Author Correction: Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins.

Giulia Pavani, Marine Laurent, Anna Fabiano

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Frequent Collaborators

5 joint publications

Guillaume Corre

4 joint publications

Annarita Miccio

3 joint publications

Oriana Romano

3 joint publications

Marine Laurent