Search Results - "Montini, E E"
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Normalization of clonal diversity in gene therapy studies using shape constrained splines
Published in Scientific reports (09-03-2022)“…Viral vectors are used to insert genetic material into semirandom genomic positions of hematopoietic stem cells which, after reinfusion into patients,…”
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Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy
Published in Human molecular genetics (15-06-2014)“…Globoid cell leukodystrophy (GLD) is an inherited lysosomal storage disease caused by β-galactocerebrosidase (GALC) deficiency. Gene therapy (GT) should…”
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Shedding of clinical-grade lentiviral vectors is not detected in a gene therapy setting
Published in Gene therapy (01-06-2015)“…Gene therapy using viral vectors that stably integrate into ex vivo cultured cells holds great promises for the treatment of monogenic diseases as well as…”
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S106: LONG‐TERM FOLLOW‐UP OF BETA‐THALASSEMIA PATIENTS TREATED WITH HEMATOPOIETIC STEM CELL GENE THERAPY
Published in HemaSphere (01-01-2022)Get full text
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Control of acromegaly in more than 90% of patients after 10 years of pegvisomant therapy: an European referral centre real-life experience
Published in Journal of endocrinological investigation (01-05-2023)“…Purpose Pegvisomant (PEG) efficaciously controls IGF-I excess in acromegaly and possesses a positive impact on glucose metabolism. Data on very prolonged PEG…”
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Cytokine-induced killer (CIK) cells engineered with chimeric antigen receptors (CARs) by sleeping beauty system
Published in Cytotherapy (Oxford, England) (01-04-2014)Get full text
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Argatroban for treatment of vein thrombosis complicating heparin-induced thrombocytopenia in haemodialyzed multiple myeloma: a case report
Published in Thrombosis research (01-04-2012)Get full text
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HIV-1 mediated insertional activation of STAT5B and BACH2 promotes the formation of a viral reservoir in T regulatory cells
Published in Journal of Virus Eradication (01-12-2017)Get full text
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HIV-1 mediated insertional activation of STAT5B promotes the formation of a viral reservoir in T regulatory cells
Published in Journal of Virus Eradication (01-12-2015)Get full text
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Relevance of an academic GMP Pan-European vector infra-structure (PEVI)
Published in Current gene therapy (01-12-2010)“…In the past 5 years, European investigators have played a major role in the development of clinical gene therapy. The provision of substantial funds by some…”
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MID2, a Homologue of the Opitz Syndrome Gene MID1: Similarities in Subcellular Localization and Differences in Expression During Development
Published in Human molecular genetics (01-08-1999)“…The B-box family is an expanding new family of genes encoding proteins involved in diverse cellular functions such as developmental patterning and oncogenesis…”
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GMP production of anti-tumor cytotoxic T-cell lines for adoptive T-cell therapy in patients with solid neoplasia
Published in Cytotherapy (Oxford, England) (2007)“…Background The adoptive transfer of ex vivo -induced tumor-specific T-cell lines provides a promising approach for cancer immunotherapy. We have demonstrated…”
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Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome
Published in Science (American Association for the Advancement of Science) (23-08-2013)“…Wiskott-Aldrich syndrome (WAS) is an inherited immunodeficiency caused by mutations in the gene encoding WASP, a protein regulating the cytoskeleton…”
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Characterisation of CTL directed towards non-inherited maternal alloantigens in human cord blood
Published in Bone marrow transplantation (Basingstoke) (01-12-1999)“…Allogeneic cord blood transplantation (CBT), especially from unrelated donors, is being increasingly used for treating paediatric patients with both malignant…”
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Solitary breast metastases from a renal cell carcinoma
Published in Breast cancer research and treatment (01-07-2001)“…A case of solitary and metachronous breast metastases from a renal cell carcinoma is described nine years after surgery. The review of the literature proves…”
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Vitamin A deficiency alters the bioelectric parameters and RNA content of rat gastric mucosa in vitro
Published in Journal of physiology and pharmacology : an official journal of the Polish Physiological Society (01-12-2003)“…The present study was aimed to investigate the mechanisms by which vitamin A plays a role in maintaining the efficiency of gastric mucosal barrier…”
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A Novel Human Serine-Threonine Phosphatase Related to the Drosophila Retinal Degeneration C (rdgC) Gene is Selectively Expressed in Sensory Neurons of Neural Crest Origin
Published in Human molecular genetics (01-07-1997)“…Through our transcriptional mapping effort in the Xp22 region, we have isolated by exon trapping a new transcript highly homologous to the Drosophila retinal…”
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Liver-directed lentiviral gene therapy in a dog model of hemophilia B
Published in Science translational medicine (04-03-2015)“…We investigated the efficacy of liver-directed gene therapy using lentiviral vectors in a large animal model of hemophilia B and evaluated the risk of…”
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AAV serotype 2 vectors preferentially integrate into active genes in mice
Published in Nature genetics (01-07-2003)“…Recombinant adeno-associated virus serotype 2 (rAAV2) is a promising vector for gene therapy because it can achieve long-term stable transgene expression in…”
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