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المؤلفون: Sheila Maestro, Gloria González-Aseguinolaza, Rafael Aldabe, Nicholas D. Weber, Nerea Zabaleta
المصدر: JHEP Reports
مصطلحات موضوعية: MPS type VII, MPSVII, S/MAR, scaffold matrix attachment regions, PB, piggyBac, gene addition, GalNAc, N-acetyl-D-galactosamine, UCD, urea cycle disorders, Genetic enhancement, medicine.medical_treatment, efficacy, MMA, methylmalonic acidemia, Review, Liver transplantation, medicine.disease_cause, MPS, mucopolysaccharidosis, immune response, IDS, iduronate 2-sulfatase, lncRNA, long non-coding RNA, gene silencing, GSD1a, glycogen storage disorder 1a, AAV, adeno-associated virus, ASOs, antisense oligonucleotides, Genome editing, AHP, acute hepatic porphyrias, HT, hereditary tyrosinemia, IMLD, inherited metabolic liver diseases, PFIC3, progressive familial cholestasis type 3, Immunology and Allergy, NHPs, non-human primates, CRISPR/Cas9, CRISPR associated protein 9, GO, glycolate oxidase, HemA/B, haemophilia A/B, Adeno-associated virus, HDR, homology-directed repair, Transcription activator-like effector nuclease, LNP, Lipid nanoparticles, siRNA, small-interfering RNA, MPR, metabolic pathway reprograming, LDH, lactate dehydrogenase, PH1, Primary hyperoxaluria type 1, gene editing, SRT, substrate reduction therapy, Gastroenterology, Ad, adenovirus, AAT, α1-antitrypsin, PA, propionic acidemia, dCas9, dead Cas9, ERT, enzyme replacement therapy, HDAd, helper-dependent adenovirus, CRISPR, clustered regularly interspaced short palindromic repeats, SB, Sleeping Beauty, LDLR, low-density lipoprotein receptor, MPS type I, MPSI, hepatocytes, OTC, ornithine transcarbamylase, APCs, antigen-presenting cells, DSBs, double-strand breaks, viral vectors, LV, lentivirus, AIP, acute intermittent porphyria, NASH, non-alcoholic steatohepatitis, GT, gene therapy, PKU, phenylketonuria, Computational biology, Gene delivery, Biology, FH, familial hypercholesterolemia, Viral vector, ITR, inverted terminal repetition, ZFN, zinc finger nucleases, GUSB, β-glucuronidase, Internal Medicine, medicine, OLT, orthotopic liver transplantation, Gene silencing, miRNAs, microRNAs, ASGPR, asialoglycoprotein receptor, ALAS1, aminolevulic synthase 1, TALEN, transcription activator-like effector nucleases, CBS, cystathionine β-synthase, PEG, polyethylene glycol, PEI, polyethyleneimine, TTR, transthyretin, Hepatology, IDUA, α-L-iduronidase, toxicity, ASGCT, American Society of Gene and Cell Therapy, CN, Crigel-Najjar, STK25, serine/threonine protein kinase 25, VLDLR, very-low-density lipoprotein receptor, WD, Wilson’s disease, FSP27, fat-specific protein 27, NHEJ, non-homologous end joining, Non-viral vectors, LTR, long terminal repeat, apoB/E, apolipoprotein B/E, RV, retrovirus, PCSK9, proprotein convertase subtilisin/kexin type 9
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::69cc33bd8f559ae9ccb64e180a7a274aTest
http://europepmc.org/articles/PMC8203845Test -
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المؤلفون: Marco Spinelli, Chiara Brombin, Nufar Marcus, Dario Di Silvestre, Patrizia Della Valle, Lucia Piceni Sereni, Maria Ester Bernardo, Pierluigi Mauri, Francesca Ferrua, Claudio Pignata, Lorella Cattaneo, Alessandro Aiuti, Lucia Dora Notarangelo, Armando D'Angelo, Marita Bosticardo, Stefania Giannelli, Koen van Rossem, Maddalena Migliavacca, Anna Villa, Loris Pozzi, Roula Farah, Maria Carmina Castiello, Maria Pia Cicalese
المساهمون: Sereni, L., Castiello, M. C., Di Silvestre, D., Della Valle, P., Brombin, C., Ferrua, Paolo, Cicalese, M. P., Pozzi, L., Migliavacca, M., Bernardo, M. E., Pignata, C., Farah, R., Notarangelo, L. D., Marcus, N., Cattaneo, L., Spinelli, M., Giannelli, S., Bosticardo, M., van Rossem, K., D'Angelo, A., Aiuti, A., Mauri, P., Villa, A.
المصدر: Journal of allergy and clinical immunology 144 (2019): 825–838. doi:10.1016/j.jaci.2019.03.012
info:cnr-pdr/source/autori:Sereni L1, Castiello MC1, Di Silvestre D2, Della Valle P3, Brombin C4, Ferrua F5, Cicalese MP6, Pozzi L3, Migliavacca M6, Bernardo ME6, Pignata C7, Farah R8, Notarangelo LD9, Marcus N10, Cattaneo L11, Spinelli M12, Giannelli S1, Bosticardo M1, van Rossem K13, D'Angelo A3, Aiuti A5, Mauri P2, Villa A14./titolo:Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome/doi:10.1016%2Fj.jaci.2019.03.012/rivista:Journal of allergy and clinical immunology/anno:2019/pagina_da:825/pagina_a:838/intervallo_pagine:825–838/volume:144
The Journal of Allergy and Clinical Immunologyمصطلحات موضوعية: 0301 basic medicine, Male, δ-g, Electron-dense granule, P-selectin, Wiskott–Aldrich syndrome, Genetic enhancement, sCD40L, Soluble CD40 ligand, vWF, von Willebrand factor, HSCT, Hematopoietic stem cell transplantation, LV, Lentivirus, 0302 clinical medicine, Immunology and Allergy, Medicine, MFI, Mean fluorescence intensity, Platelet, Child, platelet, biology, Wiskott-Aldrich syndrome, Hematopoietic Stem Cell Transplantation, X-linked thrombocytopenia, Phenotype, gene therapy, 3. Good health, 030220 oncology & carcinogenesis, Child, Preschool, platelets, Female, PRP, Platelet-rich plasma, Wiskott-Aldrich Syndrome Protein, Adult, Blood Platelets, BAFF, B cell–activating factor, Adolescent, Immunology, Article, ADP, Adenosine diphosphate, 03 medical and health sciences, CD62P, P-selectin, Von Willebrand factor, XLT, X-linked thrombocytopenia, Microscopy, Electron, Transmission, WASp, Wiskott-Aldrich syndrome protein, HMGB1, High-mobility group box 1, Humans, B-cell activating factor, GT, Gene therapy, business.industry, TEM, Transmission electron microscopy, Platelet Count, Lentivirus, FU, Follow-up, Infant, Genetic Therapy, medicine.disease, Platelet Activation, OCS, Open canalicular system, STAT3, Signal transducer and activator of transcription 3, 030104 developmental biology, CT, Closure time, GPX1, Glutathione peroxidase 1, Platelet-rich plasma, sCD62P, Soluble P-selectin, FERMT3, Fermitin family homolog 3, WAS, Wiskott-Aldrich syndrome, biology.protein, business, HD, Healthy donor, ROS, Reactive oxygen species
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8fbef2a3b9359481a3aee0ecf5da45bbTest
https://publications.cnr.it/doc/417797Test -
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المؤلفون: Paolo Uva, Mirjam van der Burg, Anna Villa, Immacolata Brigida, Francesca Ferrua, Lucia Piceni Sereni, Michael H. Albert, Giorgio Ottaviano, Marita Bosticardo, Alessandro Aiuti, Maria Carmina Castiello, Francesca Pala, Maria Grazia Roncarolo, Luigi Naldini, Samantha Scaramuzza
المساهمون: Castiello, Mc, Scaramuzza, S, Pala, F, Ferrua, F, Uva, P, Brigida, I, Sereni, L, van der Burg, M, Ottaviano, G, Albert, Mh, Grazia Roncarolo, M, Naldini, Luigi, Aiuti, Alessandro, Villa, A, Bosticardo, M., Immunology
المصدر: Journal of Allergy and Clinical Immunology, 136(3), 692-+. Mosby Inc.
The Journal of Allergy and Clinical Immunologyمصطلحات موضوعية: Male, VCN, Vector copy number, Transplantation Conditioning, Wiskott–Aldrich syndrome, medicine.medical_treatment, Gene Expression, Hematopoietic stem cell transplantation, Bone Marrow, Transduction, Genetic, B-Cell Activating Factor, Immunology and Allergy, Child, BM, Bone marrow, B cell, Wiskott-Aldrich syndrome, Hematopoietic Stem Cell Transplantation, Hematopoietic stem cell, gene therapy, 3. Good health, medicine.anatomical_structure, Child, Preschool, SDF-1α, Stromal cell–derived factor 1α, Stem cell, Wiskott-Aldrich Syndrome Protein, BAFF, B cell–activating factor, Recombinant Fusion Proteins, Genetic Vectors, Immunology, B-Lymphocyte Subsets, Immunoglobulins, Biology, primary immunodeficiency, Transplantation, Autologous, CD19, Immunophenotyping, Immune Deficiencies, Infection, and Systemic Immune Disorders, WASp, Wiskott-Aldrich syndrome protein, medicine, Humans, Progenitor cell, Autoantibodies, IVIg, Intravenous immunoglobulin, GT, Gene therapy, Gene Expression Profiling, Lentivirus, lentiviral vector, Infant, PB, Peripheral blood, Genetic Therapy, HSC, Hematopoietic stem cell, Hematopoietic Stem Cells, medicine.disease, WAS, Wiskott-Aldrich syndrome, biology.protein, Bone marrow, HD, Healthy donor
وصف الملف: application/pdf
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::495ffab946a30ddf28166ff0f448fa5aTest
https://doi.org/10.1016/j.jaci.2015.01.035Test -
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المساهمون: Kleinmann, Elodie
