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

Authors: Michela Milani, Cesare Canepari, Simone Assanelli, Simone Merlin, Ester Borroni, Francesco Starinieri, Mauro Biffi, Fabio Russo, Anna Fabiano, Desirèe Zambroni, Andrea Annoni, Luigi Naldini, Antonia Follenzi, Alessio Cantore

Source: EMBO Molecular Medicine, Vol 16, Iss 6, Pp 1427-1450 (2024)

Subject Terms: In Vivo Gene Therapy, Lentiviral Vectors, Liver Endothelial Cells, Hemophilia A, Envelope Engineering, Medicine (General), R5-920, Genetics, QH426-470

File Description: electronic resource

Relation: https://doaj.org/toc/1757-4684

Access URL: https://doaj.org/article/b045f6dbf8624a8e8e0b19badc7c0b7b

Factor VIII promotes angiogenesis and vessel stability regulating extracellular matrix proteins

Authors: Cristina Olgasi, Alessia Cucci, Ivan Molineris, Simone Assanelli, Francesca Anselmi, Chiara Borsotti, Chiara Sgromo, Andrea Lauria, Simone Merlin, Gillian E. Walker, Salvatore Oliviero, Antonia Follenzi

Source: Haematologica, Vol 999, Iss 1 (2024)

Subject Terms: Diseases of the blood and blood-forming organs, RC633-647.5

File Description: electronic resource

Relation: https://haematologica.org/article/view/11639; https://doaj.org/toc/0390-6078; https://doaj.org/toc/1592-8721

Access URL: https://doaj.org/article/3779ec1e96f04c07b5a458fdad8e794f

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

Authors: Ester Borroni, Chiara Borsotti, Roberta A. Cirsmaru, Vakhtang Kalandadze, Rosella Famà, Simone Merlin, Brian Brown, Antonia Follenzi

Source: Molecular Therapy: Nucleic Acids, Vol 35, Iss 1, Pp 102116- (2024)

Subject Terms: MT: Delivery Strategies, LSECs, immune tolerance, gene therapy, liver, transgene expression, Therapeutics. Pharmacology, RM1-950

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2162253124000039; https://doaj.org/toc/2162-2531

Access URL: https://doaj.org/article/b6e9ca40771d47899d8ded40a77f5a0f

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

Authors: Federica Esposito, Hristiana Lyubenova, Patrizia Tornabene, Stefano Auricchio, Antonella Iuliano, Edoardo Nusco, Simone Merlin, Cristina Olgasi, Giorgia Manni, Marco Gargaro, Francesca Fallarino, Antonia Follenzi, Alberto Auricchio

Source: EMBO Molecular Medicine, Vol 14, Iss 6, Pp 1-15 (2022)

Subject Terms: AAV vectors, haemophilia A, liver gene therapy, protein trans‐splicing, Medicine (General), R5-920, Genetics, QH426-470

File Description: electronic resource

Relation: https://doaj.org/toc/1757-4676; https://doaj.org/toc/1757-4684

Access URL: https://doaj.org/article/f569f4344bd64dee93d8db414a637859

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

Authors: Cristina Olgasi, Chiara Borsotti, Simone Merlin, Thorsten Bergmann, Patrick Bittorf, Adeolu Badi Adewoye, Nicholas Wragg, Kelcey Patterson, Andrea Calabria, Fabrizio Benedicenti, Alessia Cucci, Alessandra Borchiellini, Berardino Pollio, Eugenio Montini, Delfina M. Mazzuca, Martin Zierau, Alexandra Stolzing, Philip.M. Toleikis, Joris Braspenning, Antonia Follenzi

Source: Molecular Therapy: Methods & Clinical Development, Vol 23, Iss , Pp 551-566 (2021)

Subject Terms: hemophilia A, cell and gene therapy, medical device, BOEC, lentiviral vector, FVIII, Genetics, QH426-470, Cytology, QH573-671

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050121001728; https://doaj.org/toc/2329-0501

Access URL: https://doaj.org/article/7a58ac952d7247cb827b394f9103e931

Therapeutic potential of fetal liver cell transplantation in hemophilia A mice

Authors: Simone Merlin, Saicharan Akula, Alessia Cottonaro, Tamara Garcia-Leal, Luis Javier Serrano, Ester Borroni, Vakhtang Kalandadze, Rocio Galiano, Chiara Borsotti, Antonio Liras, María José Sanchez, Antonia Follenzi

Source: Haematologica, Vol 108, Iss 6 (2023)

Subject Terms: Diseases of the blood and blood-forming organs, RC633-647.5

File Description: electronic resource

Relation: https://haematologica.org/article/view/10955; https://doaj.org/toc/0390-6078; https://doaj.org/toc/1592-8721

Access URL: https://doaj.org/article/c6205903444a4a879ca23e2d551a8220

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

Authors: Patrick Bittorf, Thorsten Bergmann, Simone Merlin, Cristina Olgasi, Oliver Pullig, Ralf Sanzenbacher, Martin Zierau, Heike Walles, Antonia Follenzi, Joris Braspenning

Source: Molecular Therapy: Methods & Clinical Development, Vol 18, Iss , Pp 176-188 (2020)

Subject Terms: Cell-based therapy, gene-based therapy, ATMP, preclinical and non-clinical development, method validation, qPCR, Genetics, QH426-470, Cytology, QH573-671

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050120301169; https://doaj.org/toc/2329-0501

Access URL: https://doaj.org/article/5b6d697513714266aacd725d1db8eb11

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

Authors: Blair K. Gage, Simone Merlin, Cristina Olgasi, Antonia Follenzi, Gordon M. Keller

Source: Cell Reports, Vol 39, Iss 1, Pp 110621- (2022)

Subject Terms: hemophilia A, FVIII, liver sinusoidal endothelial cells, hPSCs, mesoderm, venous endothelial cell, Biology (General), QH301-705.5

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2211124722003692; https://doaj.org/toc/2211-1247

Access URL: https://doaj.org/article/4c3bf17d63b045ebae5e9f46262222b8

Transcriptional Targeting and MicroRNA Regulation of Lentiviral Vectors

Authors: Simone Merlin, Antonia Follenzi

Source: Molecular Therapy: Methods & Clinical Development, Vol 12, Iss , Pp 223-232 (2019)

Subject Terms: Genetics, QH426-470, Cytology, QH573-671

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050119300038; https://doaj.org/toc/2329-0501

Access URL: https://doaj.org/article/962b90333e1c4ddda9217b4352cfa648

Patient-Specific iPSC-Derived Endothelial Cells Provide Long-Term Phenotypic Correction of Hemophilia A

Authors: Cristina Olgasi, Maria Talmon, Simone Merlin, Alessia Cucci, Yvonne Richaud-Patin, Gabriella Ranaldo, Donato Colangelo, Federica Di Scipio, Giovanni N. Berta, Chiara Borsotti, Federica Valeri, Francesco Faraldi, Maria Prat, Maria Messina, Piercarla Schinco, Angelo Lombardo, Angel Raya, Antonia Follenzi

Source: Stem Cell Reports, Vol 11, Iss 6, Pp 1391-1406 (2018)

Subject Terms: Medicine (General), R5-920, Biology (General), QH301-705.5

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2213671118304351; https://doaj.org/toc/2213-6711

Access URL: https://doaj.org/article/8867c765fe344630b78a3afcd6aa23de

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

Authors: Rosella Famà, Ester Borroni, Simone Merlin, Chiara Airoldi, Silvia Pignani, Alessia Cucci, Davide Corà, Valentina Bruscaggin, Sharon Scardellato, Stefania Faletti, Giuliana Pelicci, Mirko Pinotti, Gillian E. Walker, Antonia Follenzi

Source: Haematologica, Vol 106, Iss 6 (2020)

Subject Terms: Diseases of the blood and blood-forming organs, RC633-647.5

File Description: electronic resource

Relation: https://haematologica.org/article/view/9765; https://doaj.org/toc/0390-6078; https://doaj.org/toc/1592-8721

Access URL: https://doaj.org/article/e1967f158a2648ea9ea2374d7891d731

Escape or Fight: Inhibitors in Hemophilia A

Authors: Simone Merlin, Antonia Follenzi

Source: Frontiers in Immunology, Vol 11 (2020)

Subject Terms: inhibitor, immune tolerance induction, factor VIII, hemophilia A, immune modulation, regulatory T cells, Immunologic diseases. Allergy, RC581-607

File Description: electronic resource

Relation: https://www.frontiersin.org/article/10.3389/fimmu.2020.00476/full; https://doaj.org/toc/1664-3224

Access URL: https://doaj.org/article/649794d3acbb40fd8a47a2ad2c2a8ee4

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

Authors: Isaac Canals, Jordi Soriano, Javier G. Orlandi, Roger Torrent, Yvonne Richaud-Patin, Senda Jiménez-Delgado, Simone Merlin, Antonia Follenzi, Antonella Consiglio, Lluïsa Vilageliu, Daniel Grinberg, Angel Raya

Source: Stem Cell Reports, Vol 5, Iss 4, Pp 546-557 (2015)

Subject Terms: Medicine (General), R5-920, Biology (General), QH301-705.5

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2213671115002507; https://doaj.org/toc/2213-6711

Access URL: https://doaj.org/article/41412330940d4205b3aa8f678df1978c

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

Authors: Diego Zanolini, Simone Merlin, Maria Feola, Gabriella Ranaldo, Angela Amoruso, Gianluca Gaidano, Mauro Zaffaroni, Alessandro Ferrero, Sandra Brunelleschi, Guido Valente, Sanjeev Gupta, Maria Prat, Antonia Follenzi

Source: Haematologica, Vol 100, Iss 7 (2015)

Subject Terms: Diseases of the blood and blood-forming organs, RC633-647.5

File Description: electronic resource

Relation: https://haematologica.org/article/view/7433; https://doaj.org/toc/0390-6078; https://doaj.org/toc/1592-8721

Access URL: https://doaj.org/article/d6b96a65e5f949ad80de0793a393c6c5

Human Cardiac Progenitor Spheroids Exhibit Enhanced Engraftment Potential.

Authors: Francesca Oltolina, Andrea Zamperone, Donato Colangelo, Luca Gregoletto, Simone Reano, Stefano Pietronave, Simone Merlin, Maria Talmon, Eugenio Novelli, Marco Diena, Carmine Nicoletti, Antonio Musarò, Nicoletta Filigheddu, Antonia Follenzi, Maria Prat

Source: PLoS ONE, Vol 10, Iss 9, p e0137999 (2015)

Subject Terms: Medicine, Science

File Description: electronic resource

Relation: http://europepmc.org/articles/PMC4572703?pdf=render; https://doaj.org/toc/1932-6203

Access URL: https://doaj.org/article/2224d588b376464e80bba0f141f76867

Correction: Human Cardiac Progenitor Spheroids Exhibit Enhanced Engraftment Potential.

Authors: Francesca Oltolina, Andrea Zamperone, Donato Colangelo, Luca Gregoletto, Simone Reano, Stefano Pietronave, Simone Merlin, Maria Talmon, Eugenio Novelli, Marco Diena, Carmine Nicoletti, Antonio Musarò, Nicoletta Filigheddu, Antonia Follenzi, Maria Prat

Source: PLoS ONE, Vol 10, Iss 10, p e0141632 (2015)

Subject Terms: Medicine, Science

File Description: electronic resource

Relation: http://europepmc.org/articles/PMC4619885?pdf=render; https://doaj.org/toc/1932-6203

Access URL: https://doaj.org/article/472113c6ed77489685675cdcfa747374

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].

Authors: Leonardo Badia, Andrea Baiocchi, Alfredo Todini, Simone Merlin, Silvano Pupolin, Andrea Zanella, Michele Zorzi

Source: IEEE Wireless Communications; Feb2007, Vol. 14 Issue 1, p36-43, 8p