Search Results - "Amendola, Mario"

Mechanisms and dynamics of nuclear lamina–genome interactions by Amendola, Mario, van Steensel, Bas

“…The nuclear lamina (NL) interacts with the genomic DNA and is thought to influence chromosome organization and gene expression. Both DNA sequences and histone…”
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The Cohesin Release Factor WAPL Restricts Chromatin Loop Extension by Haarhuis, Judith H.I., van der Weide, Robin H., Blomen, Vincent A., Yáñez-Cuna, J. Omar, Amendola, Mario, van Ruiten, Marjon S., Krijger, Peter H.L., Teunissen, Hans, Medema, René H., van Steensel, Bas, Brummelkamp, Thijn R., de Wit, Elzo, Rowland, Benjamin D.

“…The spatial organization of chromosomes influences many nuclear processes including gene expression. The cohesin complex shapes the 3D genome by looping…”
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Single-Cell Dynamics of Genome-Nuclear Lamina Interactions by Kind, Jop, Pagie, Ludo, Ortabozkoyun, Havva, Boyle, Shelagh, de Vries, Sandra S., Janssen, Hans, Amendola, Mario, Nolen, Leisha D., Bickmore, Wendy A., van Steensel, Bas

“…The nuclear lamina (NL) interacts with hundreds of large genomic regions termed lamina associated domains (LADs). The dynamics of these interactions and the…”
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Targeted Gene Delivery: Where to Land by Pavani, Giulia, Amendola, Mario

“…Genome-editing technologies have the potential to correct most genetic defects involved in blood disorders. In contrast to mutation-specific editing, targeted…”
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Genome-wide Maps of Nuclear Lamina Interactions in Single Human Cells by Kind, Jop, Pagie, Ludo, de Vries, Sandra S., Nahidiazar, Leila, Dey, Siddharth S., Bienko, Magda, Zhan, Ye, Lajoie, Bryan, de Graaf, Carolyn A., Amendola, Mario, Fudenberg, Geoffrey, Imakaev, Maxim, Mirny, Leonid A., Jalink, Kees, Dekker, Job, van Oudenaarden, Alexander, van Steensel, Bas

“…Mammalian interphase chromosomes interact with the nuclear lamina (NL) through hundreds of large lamina-associated domains (LADs). We report a method to map NL…”
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Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements by Lattanzi, Annalisa, Meneghini, Vasco, Pavani, Giulia, Amor, Fatima, Ramadier, Sophie, Felix, Tristan, Antoniani, Chiara, Masson, Cecile, Alibeu, Olivier, Lee, Ciaran, Porteus, Matthew H., Bao, Gang, Amendola, Mario, Mavilio, Fulvio, Miccio, Annarita

“…Editing the β-globin locus in hematopoietic stem cells is an alternative therapeutic approach for gene therapy of β-thalassemia and sickle cell disease. Using…”
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Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression by Antoniou, Panagiotis, Hardouin, Giulia, Martinucci, Pierre, Frati, Giacomo, Felix, Tristan, Chalumeau, Anne, Fontana, Letizia, Martin, Jeanne, Masson, Cecile, Brusson, Megane, Maule, Giulia, Rosello, Marion, Giovannangeli, Carine, Abramowski, Vincent, de Villartay, Jean-Pierre, Concordet, Jean-Paul, Del Bene, Filippo, El Nemer, Wassim, Amendola, Mario, Cavazzana, Marina, Cereseto, Anna, Romano, Oriana, Miccio, Annarita

“…Sickle cell disease and β-thalassemia affect the production of the adult β-hemoglobin chain. The clinical severity is lessened by mutations that cause fetal…”
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Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins by Pavani, Giulia, Laurent, Marine, Fabiano, Anna, Cantelli, Erika, Sakkal, Aboud, Corre, Guillaume, Lenting, Peter J., Concordet, Jean-Paul, Toueille, Magali, Miccio, Annarita, Amendola, Mario

“…Targeted genome editing has a great therapeutic potential to treat disorders that require protein replacement therapy. To develop a platform independent of…”
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Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy by Gentner, Bernhard, Visigalli, Ilaria, Hiramatsu, Hidefumi, Lechman, Eric, Ungari, Silvia, Giustacchini, Alice, Schira, Giulia, Amendola, Mario, Quattrini, Angelo, Martino, Sabata, Orlacchio, Aldo, Dick, John E, Biffi, Alessandra, Naldini, Luigi

“…Globoid cell leukodystrophy (GLD; also known as Krabbe disease) is an invariably fatal lysosomal storage disorder caused by mutations in the…”
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Corrigendum: Targeted Gene Delivery: Where to Land by Pavani, Giulia, Amendola, Mario

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A Double-Switch Vector System Positively Regulates Transgene Expression by Endogenous microRNA Expression (miR-ON Vector) by Amendola, Mario, Giustacchini, Alice, Gentner, Bernhard, Naldini, Luigi

“…To better understand and exploit microRNA (miR) regulation, a more precise characterization of miR expression patterns within a tissue or a lineage during…”
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Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies by Brusson, Mégane, Chalumeau, Anne, Martinucci, Pierre, Romano, Oriana, Felix, Tristan, Poletti, Valentina, Scaramuzza, Samantha, Ramadier, Sophie, Masson, Cecile, Ferrari, Giuliana, Mavilio, Fulvio, Cavazzana, Marina, Amendola, Mario, Miccio, Annarita

“…Sickle cell disease (SCD) is due to a mutation in the β-globin gene causing production of the toxic sickle hemoglobin (HbS; α2βS2). Transplantation of…”
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Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models by Lattanzi, Annalisa, Neri, Margherita, Maderna, Claudio, di Girolamo, Ilaria, Martino, Sabata, Orlacchio, Aldo, Amendola, Mario, Naldini, Luigi, Gritti, Angela

“…Leukodystrophies are rare diseases caused by defects in the genes coding for lysosomal enzymes that degrade several glycosphingolipids. Gene therapy for…”
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Oxidative stress and cell death in cells expressing L-ferritin variants causing neuroferritinopathy by Cozzi, Anna, Rovelli, Elisabetta, Frizzale, Grazia, Campanella, Alessandro, Amendola, Mario, Arosio, Paolo, Levi, Sonia

“…Abstract Neuroferritinopathies are dominantly inherited movement disorders associated with nucleotide insertions in the L-ferritin gene that modify the…”
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Author Correction: Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins by Pavani, Giulia, Laurent, Marine, Fabiano, Anna, Cantelli, Erika, Sakkal, Aboud, Corre, Guillaume, Lenting, Peter J., Concordet, Jean-Paul, Toueille, Magali, Miccio, Annarita, Amendola, Mario

“…An amendment to this paper has been published and can be accessed via a link at the top of the paper…”
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GATA Factor-Mediated Gene Regulation in Human Erythropoiesis by Romano, Oriana, Petiti, Luca, Felix, Tristan, Meneghini, Vasco, Portafax, Michel, Antoniani, Chiara, Amendola, Mario, Bicciato, Silvio, Peano, Clelia, Miccio, Annarita

“…Erythroid commitment and differentiation are regulated by the coordinated action of a host of transcription factors, including GATA2 and GATA1. Here, we…”
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Efficient Tet-Dependent Expression of Human Factor IX in Vivo by a New Self-Regulating Lentiviral Vector by Vigna, Elisa, Amendola, Mario, Benedicenti, Fabrizio, Simmons, Andrew D., Follenzi, Antonia, Naldini, Luigi

“…Regulation of gene expression represents a long-sought goal of gene therapy. However, most viral vectors pose constraints on the incorporation of…”
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Nuclear lamins are not required for lamina-associated domain organization in mouse embryonic stem cells by Amendola, Mario, van Steensel, Bas

“…In mammals, the nuclear lamina interacts with hundreds of large genomic regions, termed lamina‐associated domains (LADs) that are generally in a…”
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Easy quantitative assessment of genome editing by sequence trace decomposition by Brinkman, Eva K, Chen, Tao, Amendola, Mario, van Steensel, Bas

“…The efficacy and the mutation spectrum of genome editing methods can vary substantially depending on the targeted sequence. A simple, quick assay to accurately…”
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CRISPRthripsis: The Risk of CRISPR/Cas9-induced Chromothripsis in Gene Therapy by Amendola, Mario, Brusson, Mégane, Miccio, Annarita

“…Abstract The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 nuclease system has allowed the generation of disease models and the…”
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