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Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements
Annalisa Lattanzi
,
Vasco Meneghini
,
Giulia Pavani
,
Fatima Amor
,
Sophie Ramadier
,
et al.
Article dans une revue
hal-04325274v1
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Easy quantitative assessment of genome editing by sequence trace decomposition
Eva Brinkman
,
Tao Chen
,
Mario Amendola
,
Bas Van steensel
Article dans une revue
hal-03588465v1
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Identification of Hematopoietic Stem Cell–Specific miRNAs Enables Gene Therapy of Globoid Cell Leukodystrophy
Bernhard Gentner
,
Ilaria Visigalli
,
Hidefumi Hiramatsu
,
Eric Lechman
,
Silvia Ungari
,
et al.
Article dans une revue
hal-03588470v1
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Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models
Annalisa Lattanzi
,
Margherita Neri
,
Claudio Maderna
,
Ilaria Di Girolamo
,
Sabata Martino
,
et al.
Article dans une revue
hal-03588471v1
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Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state
Brian Brown
,
Bernhard Gentner
,
Alessio Cantore
,
Silvia Colleoni
,
Mario Amendola
,
et al.
Article dans une revue
hal-03588476v1
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Single-Cell Dynamics of Genome-Nuclear Lamina Interactions
Jop Kind
,
Ludo Pagie
,
Havva Ortabozkoyun
,
Shelagh Boyle
,
Sandra s. De vries
,
et al.
Article dans une revue
hal-03588467v1
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Targeted Gene Delivery: Where to Land
Giulia Pavani
,
Mario Amendola
Article dans une revue
hal-04407860v1
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Reprogramming T Lymphocytes for Melanoma Adoptive Immunotherapy by T-Cell Receptor Gene Transfer with Lentiviral Vectors
Sara Bobisse
,
Maria Rondina
,
Anna Merlo
,
Veronica Tisato
,
Susanna Mandruzzato
,
et al.
Article dans une revue
hal-04407866v1
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Genome-wide Maps of Nuclear Lamina Interactions in Single Human Cells
Jop Kind
,
Ludo Pagie
,
Sandra s. De vries
,
Leila Nahidiazar
,
Siddharth s. Dey
,
et al.
Article dans une revue
hal-03588463v1
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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
,
et al.
Article dans une revue
mnhn-03868573v1
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A combination of cyclophosphamide and interleukin-2 allows CD4 1 T cells converted to Tregs to control scurfy syndrome
Marianne Delville
,
Florence Bellier
,
Juliette Leon
,
Roman Klifa
,
Sabrina Lizot
,
et al.
Article dans une revue
hal-04417449v1
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The Cohesin Release Factor WAPL Restricts Chromatin Loop Extension
Judith H.I. Haarhuis
,
Robin van Der Weide
,
Vincent Blomen
,
J. Omar Yáñez-Cuna
,
Mario Amendola
,
et al.
Article dans une revue
hal-03588462v1
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The Cohesin Release Factor WAPL Restricts Chromatin Loop Extension
Judith H.I. Haarhuis
,
Robin H van der Weide
,
Vincent A Blomen
,
J. Omar Yáñez-Cuna
,
Mario Amendola
,
et al.
Article dans une revue
hal-04413676v1
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Nuclear lamins are not required for lamina‐associated domain organization in mouse embryonic stem cells
Mario Amendola
,
Bas Steensel
Article dans une revue
hal-03588464v1
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Regulated and Multiple miRNA and siRNA Delivery Into Primary Cells by a Lentiviral Platform
Mario Amendola
,
Laura Passerini
,
Ferdinando Pucci
,
Bernhard Gentner
,
Rosa Bacchetta
,
et al.
Article dans une revue
hal-03588472v1
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Nuclear lamins are not required for lamina‐associated domain organization in mouse embryonic stem cells
Mario Amendola
,
Bas van Steensel
Article dans une revue
hal-04325266v1
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GATA Factor-Mediated Gene Regulation in Human Erythropoiesis
Oriana Romano
,
Luca Petiti
,
Tristan Felix
,
Vasco Meneghini
,
Michel Portafax
,
et al.
Article dans une revue
hal-03588461v1
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Efficient Tet-Dependent Expression of Human Factor IX in Vivo by a New Self-Regulating Lentiviral Vector
Elisa Vigna
,
Mario Amendola
,
Fabrizio Benedicenti
,
Andrew Simmons
,
Antonia Follenzi
,
et al.
Article dans une revue
hal-03588478v1
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Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human b-globin locus
Chiara Antoniani
,
Vasco Meneghini
,
Annalisa Lattanzi
,
Tristan Felix
,
Oriana Romano
,
et al.
Article dans une revue
hal-04325273v1
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Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells
Giulia Pavani
,
Anna Fabiano
,
Marine Laurent
,
Fatima Amor
,
Erika Cantelli
,
et al.
Article dans une revue
hal-03588459v1
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Stable knockdown of microRNA in vivo by lentiviral vectors
Bernhard Gentner
,
Giulia Schira
,
Alice Giustacchini
,
Mario Amendola
,
Brian Brown
,
et al.
Article dans une revue
hal-03588475v1
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Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease
Sophie Ramadier
,
Anne Chalumeau
,
Tristan Felix
,
Nadia Othman
,
Sherazade Aknoun
,
et al.
Article dans une revue
hal-04416543v1
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CRISPRthripsis: The Risk of CRISPR/Cas9-induced Chromothripsis in Gene Therapy
Mario Amendola
,
Mégane Brusson
,
Annarita Miccio
Article dans une revue
hal-04325276v1
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Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human beta-globin locus
C. Antoniani
,
V. Meneghini
,
A. Lattanzi
,
T. Felix
,
O. Romano
,
et al.
Article dans une revue
hal-02880783v1
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Recent Progress in Genome Editing for Gene Therapy Applications: The French Perspective
Mario Amendola
,
Aurélie Bedel
,
Ana Buj-Bello
,
Mathieu Carrara
,
Jean-Paul Concordet
,
et al.
Article dans une revue
hal-03420183v1
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Targeted Gene Delivery: Where to Land
Giulia Pavani
,
Mario Amendola
Article dans une revue
hal-03588458v1
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Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease
Sophie Ramadier
,
Anne Chalumeau
,
Tristan Felix
,
Nadia Othman
,
Sherazade Aknoun
,
et al.
Article dans une revue
hal-04417459v1
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A Double-Switch Vector System Positively Regulates Transgene Expression by Endogenous microRNA Expression (miR-ON Vector)
Mario Amendola
,
Alice Giustacchini
,
Bernhard Gentner
,
Luigi Naldini
Article dans une revue
hal-02179338v1
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Mechanisms and dynamics of nuclear lamina–genome interactions
Mario Amendola
,
Bas van Steensel
Article dans une revue
hal-03588466v1
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Forkhead box protein 3 (FOXP3) mutations lead to increased TH17 cell numbers and regulatory T-cell instability
Laura Passerini
,
Sven Olek
,
Sara Di Nunzio
,
Federica Barzaghi
,
Sophie Hambleton
,
et al.
Article dans une revue
hal-03588469v1
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