Publications and ressources

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

Annarita Miccio , Panagiotis Antoniou, Sorana CIURA, Edor Kabashi

Source : Mol Ther

2022 jan 5

Pmid : 33823304

GATA Factor-Mediated Gene Regulation in Human Erythropoiesis.

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

Source : iScience

2020 avr 24

Pmid : 32283524

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

Annarita Miccio

Source : Sci Adv

2020 mar 5

Pmid : 32095533

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

Leslie WEBER, Giacomo Frati, Tristan Felix, Giulia Hardouin, Clara Wollenschlaeger, Vasco Meneghini, Anne Chalumeau, Marina Cavazzana, Annarita Miccio

Source : Sci Adv

2020 fév 1

Pmid : 32917636

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

Annarita Miccio , Oriana Romano

Source : IUBMB Life

2019 déc 16

Pmid : 31574210

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

Marina Cavazzana, Annarita Miccio

Source : Blood

2019 déc 13

Pmid : 31467062

Gene therapy of hemoglobinopathies: progress and future challenges.

Marina Cavazzana

Source : Hum. Mol. Genet.

2019 déc 13

Pmid : 31322165

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

Annarita Miccio

Source : Mol. Ther.

2019 déc 11

Pmid : 30424953

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

Annarita Miccio

Source : Mol Ther Methods Clin Dev

2019 nov 20

Pmid : 30140714

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

Annarita Miccio , Emmanuelle SIX, Isabelle André, Marina Cavazzana

Source : Nat Rev Drug Discov

2019 nov 8

Pmid : 30858502

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

Source : Haematologica

2019 sep 20

Pmid : 31537695

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

Source : Haematologica

2019 juil 8

Pmid : 29472357

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

Annarita Miccio

Source : Stem Cells Transl Med

2019 juin 27

Pmid : 29080249

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

Annarita Miccio , Tristan Felix

Source : Blood

2019 mai 14

Pmid : 29519807

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

Annarita Miccio

Source : Sci Rep

2017 mar 8

Pmid : 27095295