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The cohesin release factor WAPL restricts chromatin loop extension 1-gen-2017 Haarhuis, Jht; van der Weide, Rh; Blomen, Va; Yáñez-Cuna, Jo; Amendola, M; van Ruiten, Ms; Krijger, Phl; Teunissen, H; van Ruiten, Ms; Medema, Rh; van Steensel, B; Brummelkamp, Tr; de Wit, E; Rowland, Bd
GENETICALLY ENGINEERED HEMATOPOIETIC STEM CELL AS A PLATFORM FOR SYSTEMIC PROTEIN EXPRESSION 1-gen-2018 Amendola, M; Pavani, G
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus 1-gen-2018 Antoniani, C; Meneghini, V; Lattanzi, A; Felix, T; Romano, O; Magrin, E; Weber, L; Pavani, G; El Hoss, S; Kurita, R; Nakamura, Y; Cradick, Tj; Lundberg, As; Porteus, M; Amendola, M; El Nemer, W; Cavazzana, M; Mavilio, F; Miccio, A
Bifunctional vectors allowing Bcl11A silencing and expression of an anti-sickling HBB and uses thereof for gene therapy of B-hemoglobinopathies 1-gen-2019 Brusson, M; Mavilio, F; Cavazzana, M; Amendola, M; Miccio, A
Correction of beta-thalassemia phenotype by genetically engineered hematopoietic stem cells 1-gen-2019 Amendola, M; Pavani, G
Recombinant vectors suitable for the treatment of IPEX syndrome 1-gen-2019 Cavazza, M; Amendola, M; Six, E; André, I
Recombinant vectors suitable for the treatment of IPEX syndrome 1-gen-2019 Cavazza, M; Amendola, M; Six, E; André, I
Precise integration using nuclease targeted IDLV 1-gen-2019 Amendola, M; Sakkal, A; Pavani, G
Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements 1-gen-2019 Lattanzi, A; Meneghini, V; Pavani, G; Amor, F; Ramadier, S; Felix, T; Antoniani, C; Masson, C; Alibeu, O; Lee, C; Porteus, Mh; Bao, G; Amendola, M; Mavilio, F; Miccio, A
Bifunctional lentiviral vectors allowing the bs-globin silencing and expression of an anti-sickling HBB and uses thereof for gene therapy of sickle cell disease 1-gen-2020 M, Brusson; F, Mavilio; M, Cavazzana; Amendola, M; A, Miccio
Lysosomal acid lipase variants and use thereof 1-gen-2020 Amendola, M; M, Laurent
Enhancing utrophin expression in the cell by inducing mutations within the regulatory elements and therapeutics use thereof 1-gen-2020 Amendola, M; S, Guiraud
Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease 1-gen-2020 12., Weber L; Frati, G; Felix, T; Hardouin, G; Casini, A; Wollenschlaeger, C; Meneghini, V; Masson, C; De Cian, A; Chalumeau, A; Mavilio, F; Amendola, M; Andre-Schmutz, I; Cereseto, A; El Nemer, W; Concordet, Jp; Giovannangeli, C; Cavazzana, M; Miccio, A.
GATA factor-mediated gene regulation in human erythropoiesis 1-gen-2020 Romano, O; Petiti, L; Felix, T; Meneghini, V; Portafax, M; Antoniani, C; Amendola, M; Bicciato, S; Peano, C; Miccio, A
Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins 1-gen-2020 Pavani, G; Laurent, M; Fabiano, A; Cantelli, E; Sakkal, A; Corre, G; Lenting, Pj; Concordet, Jp; Toueille, M; Miccio, A; Amendola, M
Targeted gene delivery: where to land 1-gen-2021 Pavani, G; Amendola, M
Recent Progress in Genome Editing for Gene Therapy Applications: The French Perspective 1-gen-2021 Amendola, M; Bedel, A; Buj-Bello, A; Carrara, M; Concordet, Jp; Frati, G; Gilot, D; Giovannangeli, C; Gutierrez-Guerrero, A; Laurent, M; Miccio, A; Moreau-Gaudry, F; Sourd, C; Valton, J; Verhoeyen, E.
A combination of cyclophosphamide, interleukin-2 allow CD4+ T cells converted to Tregs to control scurfy syndrome. 1-gen-2021 8., Delville M; Bellier, F; Leon, J; Klifa, R; Lizot, S; Vinçon, H; Sobrino, S; Thouenon, R; Marchal, A; Garrigue, A; Olivre, J; Charbonnier, S; Lagresle-Peyrou, C; Amendola, M; Schambach, A; Gross, Da; Lamarthée, B; Benoist, C; Zuber, J; André, I; Cavazzana, M; Six, E
Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells 1-gen-2021 7., Pavani G; Fabiano, A; Laurent, M; Amor, F; Cantelli, E; Chalumeau, A; Concordet, Jp; Mavilio, F; Ferrari, G.; Miccio, A; Amendola, M
Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease 1-gen-2022 5., Ramadier S; Chalumeau, A; Felix, T; Othman, N; Aknoun, S; Casini, A; Maule, G; Masson, C; De Cian, A; Frati, G; Brusson, M; Concordet, Jp; Cavazzana, M; Cereseto, A; El Nemer, W; Amendola, M; Wattellier, B; Meneghini, V; Miccio, A.
Mostrati risultati da 21 a 40 di 41
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