Publications and ressources

Base and Prime Editing Technologies for Blood Disorders.

Panagiotis Antoniou, Annarita Miccio , Mégane Brusson

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A Genome Editing System for Therapeutical Targeting of Stem Cells.

Giacomo Frati, Annarita Miccio

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GATA Factor-Mediated Gene Regulation in Human Erythropoiesis.

Oriana Romano, Tristan Felix, Vasco Meneghini, Chiara Antoniani, Annarita Miccio

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

Annarita Miccio

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GATA factor transcriptional activity: Insights from genome-wide binding profiles.

Annarita Miccio , Oriana Romano

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Gene therapy of hemoglobinopathies: progress and future challenges.

Marina Cavazzana

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Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies.

Marina Cavazzana, Annarita Miccio

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Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements.

Annarita Miccio

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An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype.

Annarita Miccio

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Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges.

Annarita Miccio , Emmanuelle SIX, Isabelle André, Marina Cavazzana

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Extensive multilineage analysis in patients with mixed chimerism after allogeneic transplantation for sickle cell disease: insight into hematopoiesis and engraftment thresholds for gene therapy.

Marina Cavazzana

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Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion.

Chantal LAGRESLE-PEYROU, Oriana Romano, Tristan Felix, Marianne DELVILLE, Isabelle André, Marina Cavazzana, Annarita Miccio , Tristan Felix

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Concise Review: Epigenetic Regulation of Hematopoiesis: Biological Insights and Therapeutic Applications.

Annarita Miccio

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Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus.

Annarita Miccio , Tristan Felix

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Transcriptional, epigenetic and retroviral signatures identify regulatory regions involved in hematopoietic lineage commitment.

Annarita Miccio

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