Publications and ressources

Revolutionising healing: Gene Editing's breakthrough against sickle cell disease.

Annarita Miccio

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Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi.

Annarita Miccio

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Sickle Cell Disease: From Genetics to Curative Approaches.

Giulia Hardouin, Marina Cavazzana, Annarita Miccio

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

Mégane Brusson, Anne Chalumeau, Pierre Martinucci, Oriana Romano, Tristan Felix, Sophie Ramadier, Marina Cavazzana, Annarita Miccio

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Adenine base editor-mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation.

Giulia Hardouin, Panagiotis Antoniou, Pierre Martinucci, Tristan Felix, Marina Cavazzana, Annarita Miccio

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Epigenetic Regulation of β-Globin Genes and the Potential to Treat Hemoglobinopathies through Epigenome Editing.

Letizia Fontana, Annarita Miccio , Panagiotis Antoniou

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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, Giacomo Frati, Tristan Felix, Anne Chalumeau, Letizia Fontana, Jeanne Martin, Mégane Brusson, Vincent Abramowski, Jean-Pierre De Villartay, Marina Cavazzana, Oriana Romano, Annarita Miccio

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CRISPRthripsis: The Risk of CRISPR/Cas9-induced Chromothripsis in Gene Therapy.

Mégane Brusson, Annarita Miccio

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Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial.

Anne Chalumeau, Adeline DENIS, Bénédicte Neven, Emmanuelle SIX, Annarita Miccio , Marina Cavazzana

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Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease.

Sophie Ramadier, Anne Chalumeau, Tristan Felix, Giacomo Frati, Mégane Brusson, Marina Cavazzana, Vasco Meneghini, Annarita Miccio

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Novel genome-editing-based approaches to treat motor neuron diseases: Promises and challenges.

Annarita Miccio , Panagiotis Antoniou, Sorana CIURA, Edor Kabashi

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Fetal hemoglobin rescues ineffective erythropoiesis in sickle cell disease.

Michael Dussiot, Giacomo Frati, Annarita Miccio

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Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells.

Anne Chalumeau, Annarita Miccio

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Genome editing approaches to β-hemoglobinopathies.

Mégane Brusson, Annarita Miccio

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Genome Editing for β-Hemoglobinopathies: Advances and Challenges.

Giacomo Frati, Annarita Miccio

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