Výsledky vyhledávání - "Leslie Weber"

Akademický článek

An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype

Autor: Leslie Weber, Valentina Poletti, Elisa Magrin, Chiara Antoniani, Samia Martin, Charles Bayard, Hanem Sadek, Tristan Felix, Vasco Meneghini, Michael N. Antoniou, Wassim El-Nemer, Fulvio Mavilio, Marina Cavazzana, Isabelle Andre-Schmutz, Annarita Miccio

Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 10, Iss , Pp 268-280 (2018)

Autologous transplantation of hematopoietic stem cells transduced with a lentiviral vector (LV) expressing an anti-sickling HBB variant is a potential treatment for sickle cell disease (SCD). With a clinical trial as our ultimate goal, we generated L

Externí odkaz: https://doaj.org/article/86a442d8230844db8aa5394698431fd8

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Akademický článek

Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion

Publikováno v: Haematologica, Vol 103, Iss 5 (2018)

Sickle cell disease is characterized by chronic anemia and vaso-occlusive crises, which eventually lead to multi-organ damage and premature death. Hematopoietic stem cell transplantation is the only curative treatment but it is limited by toxicity an

Externí odkaz: https://doaj.org/article/38e6828b68394087a1a5f54437c70e29

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Editing a y-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

Autor: Anne Chalumeau, Cécile Masson, Marina Cavazzana, Tristan Felix, Vasco Meneghini, Anne De Cian, Isabelle André-Schmutz, Anna Cereseto, Wassim El Nemer, Giacomo Frati, Carine Giovannangeli, Clara Wollenschlaeger, Leslie Weber, Mario Amendola, Giulia Hardouin, Antonio Casini, Fulvio Mavilio, Annarita Miccio, Jean-Paul Concordet

Publikováno v: Science Advances
Science Advances, American Association for the Advancement of Science (AAAS), 2020, 6 (7), pp.eaay9392. ⟨10.1126/sciadv.aay9392⟩

Editing the fetal γ-globin promoters in hematopoietic stem cells from sickle cell disease patients induces therapeutic γ-globin levels.
Sickle cell disease (SCD) is caused by a single amino acid change in the adult hemoglobin (Hb) β chain tha

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f821ecb9947713ce7c6b250a43b2e0d1
https://hal-mnhn.archives-ouvertes.fr/mnhn-03100446

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Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human beta-globin locus

Autor: Giulia Pavani, Wassim El Nemer, Oriana Romano, Chiara Antoniani, Ante Sven Lundberg, Yukio Nakamura, Vasco Meneghini, Sara El Hoss, Fulvio Mavilio, Annarita Miccio, Annalisa Lattanzi, Thomas J. Cradick, Mario Amendola, Marina Cavazzana, Ryo Kurita, Tristan Felix, Matthew H. Porteus, Elisa Magrin, Leslie Weber

Publikováno v: Blood
Blood, American Society of Hematology, 2018, 131, pp.1960-1973. ⟨10.1182/blood-2017-10-811505⟩
Blood, 2018, 131, pp.1960-1973. ⟨10.1182/blood-2017-10-811505⟩

International audience; Naturally occurring, large deletions in the beta-globin locus result in hereditary persistence of fetal hemoglobin, a condition that mitigates the clinical severity of sickle cell disease (SCD) and beta-thalassemia. We designe

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b4b03950f429c7272d83511e6c369566
https://hal.archives-ouvertes.fr/hal-02880783

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Gene Therapy in a Patient with Sickle Cell Disease

Publikováno v: New England Journal of Medicine
New England Journal of Medicine, Massachusetts Medical Society, 2017, 376 (9), pp.848-855. ⟨10.1056/NEJMoa1609677⟩
NEW ENGLAND JOURNAL OF MEDICINE
r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
Universidad de las Islas Baleares
New England Journal of Medicine, 2017, 376 (9), pp.848-855. ⟨10.1056/NEJMoa1609677⟩

Sickle cell disease results from a homozygous missense mutation in the ß-globin gene that causes polymerization of hemoglobin S. Gene therapy for patients with this disorder is complicated by the complex cellular abnormalities and challenges in achi

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6ee6a056a57a254d6647a300aac19bf8
https://www.hal.inserm.fr/inserm-01834781/document

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Species identification by peptide mass fingerprinting (PMF) in fibre products preserved by association with copper-alloy artefacts

Autor: Caroline Solazzo, Leslie Weber, Penelope Walton Rogers, Julie Wilson, Harriet F. Beaubien, Matthew J. Collins

Publikováno v: Journal of Archaeological Science. 49:524-535

Fibre products, such as textiles and animal pelts, are often recovered in the corrosion crust of archaeological metal artefacts. Because clothed burials are an important resource for the study of past societies, accurate fibre identification is impor

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::d938fe5433a2968d66c5fb88d66ecfc0
https://doi.org/10.1016/j.jas.2014.06.009

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559. Induction of Fetal Hemoglobin in Adult Erythroblasts by Genome Editing of the Beta-Globin Locus

Autor: Giulia Pavani, Fulvio Mavilio, Leslie Weber, Elisa Magrin, Matthew H. Porteus, Chiara Antoniani, Annalisa Lattanzi, Annarita Miccio, Marina Cavazzana, Vasco Meneghini

Publikováno v: Molecular Therapy. 24:S223-S224

Sickle cell disease (SCD) and β-thalassemia are severe anemias characterized by abnormal or reduced production of hemoglobin β-chains. Autologous transplantation of genetically corrected hematopoietic stem cells (HSC) is an attractive therapeutic a

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::7abf26d0076d8b42d75e9e258116b35e
https://doi.org/10.1016/s1525-0016(16)33367-6

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