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Escape or Fight: Inhibitors in Hemophilia A

Frontiers in immunology, 2020-03, Vol.11, p.476-476

2020

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Factor VIII promotes angiogenesis and vessel stability regulating extracellular matrix proteins

Haematologica (Roma), 2024-10, Vol.109 (10), p.3391-3397

2024

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Transcriptional Targeting and MicroRNA Regulation of Lentiviral Vectors

Molecular therapy. Methods & clinical development, 2019-03, Vol.12, p.223-232

2019

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Therapeutic correction of hemophilia A by transplantation of hPSC-derived liver sinusoidal endothelial cell progenitors

Cell reports (Cambridge), 2022-04, Vol.39 (1), p.110621-110621, Article 110621

2022

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GP64-pseudotyped lentiviral vectors target liver endothelial cells and correct hemophilia A mice

EMBO molecular medicine, 2024-06, Vol.16 (6), p.1427-1450

2024

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Role of bone marrow transplantation for correcting hemophilia A in mice

Blood, 2012-06, Vol.119 (23), p.5532-5542

2012

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Therapeutic potential of fetal liver cell transplantation in hemophilia A mice

Haematologica (Roma), 2023-06, Vol.108 (6), p.1544-1554

2023

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Immune tolerance promotion by LSEC-specific lentiviral vector-mediated expression of the transgene regulated by the stabilin-2 promoter

Molecular therapy. Nucleic acids, 2024-03, Vol.35 (1), p.102116-102116, Article 102116

2024

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Deciphering the Ets-1/2-mediated transcriptional regulation of F8 gene identifies a minimal F8 promoter for hemophilia A gene therapy

Haematologica (Roma), 2021-06, Vol.106 (6), p.1624-1635

2021

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Liver gene therapy with intein‐mediated F8 trans‐splicing corrects mouse haemophilia A

EMBO molecular medicine, 2022-06, Vol.14 (6), p.e15199-n/a

2022

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Extrahepatic sources of factor VIII potentially contribute to the coagulation cascade correcting the bleeding phenotype of mice with hemophilia A

Haematologica (Roma), 2015-07, Vol.100 (7), p.881-892

2015

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Human Cardiac Progenitor Spheroids Exhibit Enhanced Engraftment Potential

PloS one, 2015-09, Vol.10 (9), p.e0137999

2015

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Kupffer Cell Transplantation in Mice for Elucidating Monocyte/Macrophage Biology and for Potential in Cell or Gene Therapy

The American journal of pathology, 2016-03, Vol.186 (3), p.539-551

2016

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Diacylglycerol kinases are essential for hepatocyte growth factor‐dependent proliferation and motility of Kaposi’s sarcoma cells

Cancer science, 2011-07, Vol.102 (7), p.1329-1336

2011

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Dissecting the transcriptional phenotype of ribosomal protein deficiency: implications for Diamond-Blackfan Anemia

Gene, 2014-07, Vol.545 (2), p.282-289

2014

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Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device

Molecular therapy. Methods & clinical development, 2021-12, Vol.23, p.551-566

2021

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Regulatory-Compliant Validation of a Highly Sensitive qPCR for Biodistribution Assessment of Hemophilia A Patient Cells

Molecular therapy. Methods & clinical development, 2020-09, Vol.18, p.176-188

2020

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Deletion of the ectodomain unleashes the transforming, invasive, and tumorigenic potential of the MET oncogene

Cancer science, 2009-04, Vol.100 (4), p.633-638

2009

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Activity and High-Order Effective Connectivity Alterations in Sanfilippo C Patient-Specific Neuronal Networks

Stem cell reports, 2015-10, Vol.5 (4), p.546-557

2015

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Dendritic cell-mediated in vivo bone resorption

The Journal of immunology (1950), 2010-08, Vol.185 (3), p.1485-1491

2010

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Correction: Human Cardiac Progenitor Spheroids Exhibit Enhanced Engraftment Potential

PloS one, 2015, Vol.10 (10), p.e0141632-e0141632

2015

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A long term, non-tumorigenic rat hepatocyte cell line and its malignant counterpart, as tools to study hepatocarcinogenesis

Oncotarget, 2017-02, Vol.8 (9), p.15716-15731

2017

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On the impact of physical layer awareness on scheduling and resource allocation in broadband multicellular IEEE 802.16 systems [Radio Resource Management and Protocol Engineering for IEEE 802.16]

IEEE wireless communications, 2007-02, Vol.14 (1), p.36-43

2007

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Agonist monoclonal antibodies against HGF receptor protect cardiac muscle cells from apoptosis

American journal of physiology. Heart and circulatory physiology, 2010-04, Vol.298 (4), p.H1155-H1165

2010

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Durable and efficient gene silencing in vivo by hit-and-run epigenome editing

Nature (London), 2024-03, Vol.627 (8003), p.416-423

2024

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Escape or Fight: Inhibitors in Hemophilia A

Factor VIII promotes angiogenesis and vessel stability regulating extracellular matrix proteins

Transcriptional Targeting and MicroRNA Regulation of Lentiviral Vectors

Therapeutic correction of hemophilia A by transplantation of hPSC-derived liver sinusoidal endothelial cell progenitors

GP64-pseudotyped lentiviral vectors target liver endothelial cells and correct hemophilia A mice

Role of bone marrow transplantation for correcting hemophilia A in mice

Therapeutic potential of fetal liver cell transplantation in hemophilia A mice

Immune tolerance promotion by LSEC-specific lentiviral vector-mediated expression of the transgene regulated by the stabilin-2 promoter

Deciphering the Ets-1/2-mediated transcriptional regulation of F8 gene identifies a minimal F8 promoter for hemophilia A gene therapy

Liver gene therapy with intein‐mediated F8 trans‐splicing corrects mouse haemophilia A

Extrahepatic sources of factor VIII potentially contribute to the coagulation cascade correcting the bleeding phenotype of mice with hemophilia A

Human Cardiac Progenitor Spheroids Exhibit Enhanced Engraftment Potential

Kupffer Cell Transplantation in Mice for Elucidating Monocyte/Macrophage Biology and for Potential in Cell or Gene Therapy

Diacylglycerol kinases are essential for hepatocyte growth factor‐dependent proliferation and motility of Kaposi’s sarcoma cells

Dissecting the transcriptional phenotype of ribosomal protein deficiency: implications for Diamond-Blackfan Anemia

Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device

Regulatory-Compliant Validation of a Highly Sensitive qPCR for Biodistribution Assessment of Hemophilia A Patient Cells

Deletion of the ectodomain unleashes the transforming, invasive, and tumorigenic potential of the MET oncogene

Activity and High-Order Effective Connectivity Alterations in Sanfilippo C Patient-Specific Neuronal Networks

Dendritic cell-mediated in vivo bone resorption

Correction: Human Cardiac Progenitor Spheroids Exhibit Enhanced Engraftment Potential

A long term, non-tumorigenic rat hepatocyte cell line and its malignant counterpart, as tools to study hepatocarcinogenesis

On the impact of physical layer awareness on scheduling and resource allocation in broadband multicellular IEEE 802.16 systems [Radio Resource Management and Protocol Engineering for IEEE 802.16]

Agonist monoclonal antibodies against HGF receptor protect cardiac muscle cells from apoptosis

Durable and efficient gene silencing in vivo by hit-and-run epigenome editing

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