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A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders

Autor: Francesca Tucci, Stefania Galimberti, Luigi Naldini, Maria Grazia Valsecchi, Alessandro Aiuti

Ex-vivo gene therapy (GT) with hematopoietic stem and progenitor cells (HSPCs) engineered with integrating vectors is a promising treatment for monogenic diseases, but lack of centralized databases is hampering an overall outcomes assessment. Here we

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::00425b786c2b4650c4f8222c39f88dd5
http://hdl.handle.net/10281/372387

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Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency

Autor: Annaliesse Blincoe, Alessandro Aiuti, Luigi D. Notarangelo, Michael S. Hershfield, H. Bobby Gaspar, Jennifer M. Puck, Donald B. Kohn, Eyal Grunebaum

Publikováno v: The Journal of allergy and clinical immunology, vol 143, iss 3

Inherited defects in adenosine deaminase (ADA) cause a subtype of severe combined immunodeficiency (SCID) known as severe combined immune deficiency caused by adenosine deaminase defects (ADA-SCID). Most affected infants can receive a diagnosis while

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::330b303d248e99dda93342c6d19dce03
https://doi.org/10.1016/j.jaci.2018.08.024

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Oncogene-induced senescence in hematopoietic progenitors features myeloid restricted hematopoiesis, chronic inflammation and histiocytosis

Publikováno v: Nature Communications
Nature Communications, Vol 12, Iss 1, Pp 1-18 (2021)

Activating mutations in the BRAF-MAPK pathway have been reported in histiocytoses, hematological inflammatory neoplasms characterized by multi-organ dissemination of pro-inflammatory myeloid cells. Here, we generate a humanized mouse model of transpl

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::05652830ea4b45f2383d98efb5f8e1bb

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Direct Cell Reprogramming of Mouse Fibroblasts into Functional Astrocytes Using Lentiviral Overexpression of the Transcription Factors NFIA, NFIB, and SOX9

Autor: Qiu, Boning, de Vries, Ruben J, Caiazzo, Massimiliano, Ahlenius, Henrik, Afd Pharmaceutics, Pharmaceutics

Publikováno v: Methods in Molecular Biology ISBN: 9781071616000
Neural Reprogramming, 2352, 31. Springer

Astrocytes play an important role in maintaining brain homeostasis and their dysfunction is involved in a number of neurological disorders. An accessible source of astrocytes is essential to model neurological diseases and potential cell therapy appr

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0752876eb656081592e9ae392740d5b9
https://doi.org/10.1007/978-1-0716-1601-7_3

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Murine Rankl−/− Mesenchymal Stromal Cells Display an Osteogenic Differentiation Defect Improved by a RANKL-Expressing Lentiviral Vector

Autor: Valentina Capo, Marco Gattorno, Cristina Sobacchi, Ileana Bortolomai, Ciro Menale, Anna Tampieri, Elisabetta Traggiai, Lorenzo Diomede, Camilla Recordati, Francesca Schena, Monica Sandri, Emanuela Caci, Eleonora Palagano, Arinna Bertoni, Alberto Martini, Claudia Pastorino, Anna Villa

Publikováno v: Stem Cells. 35:1365-1377

Autosomal recessive osteopetrosis (ARO) is a severe bone disease characterized by increased bone density due to impairment in osteoclast resorptive function or differentiation. Hematopoietic stem cell transplantation is the only available treatment;

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::72f6751aa5f1ad1b42889c663b6d1ae6
https://doi.org/10.1002/stem.2574

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Assessing the Impact of Cyclosporin A on Lentiviral Transduction and Preservation of Human Hematopoietic Stem Cells in Clinically Relevant Ex Vivo Gene Therapy Settings

Autor: Francesco Piras, Carolina Petrillo, Alessia Capotondo, Eugenio Montini, Andrea Calabria, Bernhard Gentner, Ivan Cuccovillo, Giulio Spinozzi, Anna Kajaste-Rudnitski, Luigi Naldini, Fabrizio Benedicenti, Alessandra Biffi

Publikováno v: Human Gene Therapy

Improving hematopoietic stem and progenitor cell (HSPC) permissiveness to lentiviral vector (LV) transduction without compromising their biological properties remains critical for broad-range implementation of gene therapy as a treatment option for s

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::17da3373fd6061ca25f5a0bbeac26d3b
http://hdl.handle.net/11577/3317963

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Non-primate lentiviral vectors and their applications in gene therapy for ocular disorders

Autor: Elena Baiamonte, Melania Lo Iacono, Vincenzo Cavalieri

Publikováno v: Viruses, Vol 10, Iss 6, p 316 (2018)
Viruses

Lentiviruses have a number of molecular features in common, starting with the ability to integrate their genetic material into the genome of non-dividing infected cells. A peculiar property of non-primate lentiviruses consists in their incapability t

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::eaf401eb8a55f10ed34d34c5b9dff09c
http://hdl.handle.net/10447/365079

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Dynamics of genetically engineered hematopoietic stem and progenitor cells after autologous transplantation in humans

Autor: Francesca Ferrua, Alessandro Aiuti, Luca Biasco, Serena Scala, Luca Basso-Ricci, Stefania Giannelli, Maria Pia Cicalese, Francesca Dionisio, Federica Andrea Salerio, Lorena Leonardelli, Danilo Pellin

Hematopoietic stem and progenitor cells (HSPC) are endowed with the role of generating and maintaining lifelong the extremely diverse pool of blood cells(1). Clinically, transplantation of human HSPC from an allogeneic healthy donor or infusion of au

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6912cd34659e1d93ae26441c3ca16324
http://www.nature.com/nm/index.html

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Site-Specific Direct Labeling of Neurotrophins and Their Receptors: From Biochemistry to Advanced Imaging Applications

Autor: Fulvio Bonsignore, Laura Marchetti, Rosy Amodeo, Antonino Cattaneo, Francesco Gobbo

Publikováno v: Neurotrophic Factors ISBN: 9781493975709

We describe here a versatile methodological platform to achieve site-directed and stoichiometry-controlled labeling of neurotrophins and their receptors with various probes, ranging from biotin to small organic dyes. This labeling method works in vit

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2f2d1908dfc9850941ab4c38e5f668e0
https://doi.org/10.1007/978-1-4939-7571-6_22

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Efficient Ex Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy

Autor: Giuliana Ferrari, Luigi Naldini, Maria Rosa Lidonnici, Francesco Boccalatte, Erika Zonari, Anna Kajaste-Rudnitski, Bernhard Gentner, Giacomo Desantis, Alessandro Aiuti, Carolina Petrillo

Publikováno v: Stem Cell Reports, Vol 8, Iss 4, Pp 977-990 (2017)
Stem Cell Reports

Summary Ex vivo gene therapy based on CD34+ hematopoietic stem cells (HSCs) has shown promising results in clinical trials, but genetic engineering to high levels and in large scale remains challenging. We devised a sorting strategy that captures mor

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::554c908fc973bae538531a477a1fb640
http://www.elsevier.com/journals/stem-cell-reports/2213-6711

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