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  • 1
    Language: English
    In: Bone marrow transplantation (Basingstoke), 2008-10, Vol.42 (S2), p.S2-S6
    Description: Nowadays, different treatment options are available for an extending list of lysosomal storage diseases (LSDs). Hematopoietic stem cell transplantation (HSCT) can benefit selected subsets of patients with some LSDs, but results have been poor in several other disorders, including metachromatic leukodystrophy (MLD), outlining the need for innovative therapeutic approaches in this field. Enzyme replacement therapy has been developed recently for MLD, and a Phase I/II trial is ongoing. However, the blood-brain barrier limits the access of the recombinant product to the nervous tissues. Autologous hematopoietic stem/progenitor cells can be genetically modified to constitutively express supra-physiological levels of arylsulfatase-A and may become a quantitatively more effective source of functional enzyme than normal donor cells when transplanted in patients with MLD, thus possibly overcoming the limits of HSCT. Moreover, autologous transplantation might be associated with a significantly reduced transplant-related morbidity and TRM avoiding the risk of GVHD. Therefore, such a gene therapy strategy could represent a significant advance in comparison to conventional allogeneic HSCT.
    Subject(s): Recombinant Proteins - therapeutic use ; Hematopoietic Stem Cell Transplantation - trends ; Humans ; Risk Factors ; Leukodystrophy, Metachromatic - therapy ; Transplantation, Autologous ; Genetic Therapy - trends ; Transplantation, Homologous ; Graft vs Host Disease ; Cerebroside-Sulfatase - therapeutic use ; Blood-Brain Barrier - enzymology ; Leukodystrophy, Metachromatic - enzymology ; Clinical Trials, Phase I as Topic ; Clinical Trials, Phase II as Topic ; Metachromatic leukodystrophy ; Care and treatment ; Usage ; Patient outcomes ; Stem cells ; Transplantation ; Gene therapy ; Index Medicus
    ISSN: 0268-3369
    E-ISSN: 1476-5365
    Source: Nature Open Access
    Source: Academic Search Ultimate
    Source: Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals
    Source: Alma/SFX Local Collection
    Source: © ProQuest LLC All rights reserved〈img src="https://exlibris-pub.s3.amazonaws.com/PQ_Logo.jpg" style="vertical-align:middle;margin-left:7px"〉
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  • 2
    Language: English
    In: Bone marrow transplantation (Basingstoke), 2008-06, Vol.41 (S2), p.S87-S89
    Description: Haematopoietic cell transplantation (HCT) can benefit some selected subsets of patients with lysosomal diseases. Results had been impressive in children with MPS I-H, but poor in other disorders. Careful, multidisciplinary decision-making regarding whether to recommend HCT and how to provide optimal pre- and post-HCT care has proven essential to increase the likelihood of a good outcome.
    Subject(s): Lysosomal Storage Diseases - therapy ; Humans ; Child, Preschool ; Hematopoietic Stem Cell Transplantation ; Infant ; Mesenchymal Stem Cell Transplantation ; Cord Blood Stem Cell Transplantation ; Metabolism, Inborn errors of ; Enzymes ; Care and treatment ; Transplantation ; Diagnosis ; Research ; Health aspects ; Hematopoietic stem cells ; Index Medicus
    ISSN: 0268-3369
    E-ISSN: 1476-5365
    Source: Nature Open Access
    Source: Academic Search Ultimate
    Source: Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals
    Source: Alma/SFX Local Collection
    Source: © ProQuest LLC All rights reserved〈img src="https://exlibris-pub.s3.amazonaws.com/PQ_Logo.jpg" style="vertical-align:middle;margin-left:7px"〉
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  • 3
    Language: English
    In: Pure and applied geophysics, 2014-11, Vol.171 (11), p.3083-3097
    Description: Ambient noise measurements performed on the western flank of Mt. Etna are analyzed to infer the occurrence of directional amplification effects in fault zones. The data were recorded along short (〈500 m) profiles crossing the Ragalna Fault System. Ambient noise records were processed to compute the horizontal-to-vertical noise spectral ratio as a function of frequency and direction of motion. Wavefield polarization was investigated in the time–frequency domain as well. Peaks of the spectral ratios generally fall in the frequency band 1.0–6.0 Hz pointing out directional amplifications that are also confirmed by the results of the time–frequency analysis, the largest amplification occurring with high angle to the fault strike. A variation of the frequency of the spectral peak is observed between the two sides of the fault, possibly related to a damage fault asymmetry. Measurements performed several kilometers away from the fault zone do not show behavior that is as systematic as in the fault zone, and this suggests that the observed directional effects can be ascribed to the fault fabric. We relate the polarization effect to compliance anisotropy in the fault zone, where the presence of predominantly oriented fractures makes the normal component of ground motion larger than the transversal one. In order to test the direction and the type of fractures that are expected in the fault zone, we modeled the brittle deformation pattern of the investigated fault. Theoretical results are in good agreement with field observations of the fracture strike.
    Subject(s): Earth Sciences ; Directional resonance ; Geophysics/Geodesy ; fault zone ; Mt. Etna ; wavefield polarization ; Models ; Seismology ; Anisotropy ; Analysis
    ISSN: 0033-4553
    E-ISSN: 1420-9136
    Source: Academic Search Ultimate
    Source: Alma/SFX Local Collection
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  • 4
    Language: English
    In: Bone marrow transplantation (Basingstoke), 2017-12, Vol.52 (12), p.1684-1685
    Subject(s): Index Medicus
    ISSN: 0268-3369
    E-ISSN: 1476-5365
    Source: Nature Open Access
    Source: Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals
    Source: Alma/SFX Local Collection
    Source: © ProQuest LLC All rights reserved〈img src="https://exlibris-pub.s3.amazonaws.com/PQ_Logo.jpg" style="vertical-align:middle;margin-left:7px"〉
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  • 5
    Language: English
    In: Bone marrow transplantation (Basingstoke), 2011-07, Vol.46 (7), p.987-992
    Description: Progressive multifocal leukoencephalopathy (PML) associated with polyomavirus JC (JCV) infection has been reported to be usually fatal in allogeneic hematopoietic SCT (HSCT) recipients. We present the case of a 19-year-old HSCT patient diagnosed with JCV-associated PML after prolonged immunosuppression for severe GVHD. No short-term neurological improvement was observed after antiviral treatment and discontinuation of immunosuppressive therapy. Donor-derived JCV Ag-specific CTLs were generated in vitro after stimulation with 15-mer peptides derived from VP1 and large T viral proteins. After adoptive CTL infusion, virus-specific cytotoxic cells were shown in the peripheral blood, JCV-DNA was cleared in the cerebrospinal fluid and the patient showed remarkable improvement. Adoptive T-lymphocyte therapy with JCV-specific CTLs was feasible and had no side effects. This case suggests that adoptive transfer of JCV-targeted CTLs may contribute to restore JCV-specific immune competence and control PML in transplanted patients.
    Subject(s): Infectious diseases ; Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy ; Bone marrow, stem cells transplantation. Graft versus host reaction ; Viral diseases ; Transfusions. Complications. Transfusion reactions. Cell and gene therapy ; Biological and medical sciences ; Viral diseases of the nervous system ; Medical sciences ; Human viral diseases ; Leukoencephalopathy, Progressive Multifocal - immunology ; T-Lymphocytes, Cytotoxic - immunology ; Young Adult ; Leukoencephalopathy, Progressive Multifocal - therapy ; Immunotherapy, Adoptive - methods ; JC Virus - immunology ; Humans ; Adolescent ; Hematopoietic Stem Cell Transplantation - adverse effects ; Leukoencephalopathy, Progressive Multifocal - virology ; Adult ; Male ; Case studies ; Care and treatment ; Physiological aspects ; Development and progression ; Transplantation ; Diagnosis ; T cells ; Leukoencephalopathy ; Health aspects ; Hematopoietic stem cells ; Index Medicus
    ISSN: 0268-3369
    E-ISSN: 1476-5365
    Source: Nature Open Access
    Source: Academic Search Ultimate
    Source: Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals
    Source: Alma/SFX Local Collection
    Source: © ProQuest LLC All rights reserved〈img src="https://exlibris-pub.s3.amazonaws.com/PQ_Logo.jpg" style="vertical-align:middle;margin-left:7px"〉
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  • 6
    Language: English
    In: Bone marrow transplantation (Basingstoke), 2015-02, Vol.50 (2), p.181-188
    Description: We analyzed the outcome of 243 children with high-risk (HR) AML in first CR1 enrolled in the AIEOP-2002/01 protocol, who were given either allogeneic (ALLO; n=141) or autologous (AUTO; n=102) hematopoietic SCT (HSCT), depending on the availability of a HLA-compatible sibling. Infants, patients with AML-M7, or complex karyotype or those with FLT3-ITD, were eligible to be transplanted also from alternative donors. All patients received a myeloablative regimen combining busulfan, cyclophosphamide and melphalan; [corrected] AUTO-HSCT patients received BM cells in most cases, while in children given ALLO-HSCT stem cell source was BM in 96, peripheral blood in 19 and cord blood in 26. With a median follow-up of 57 months (range 12-130), the probability of disease-free survival (DFS) was 73% and 63% in patients given either ALLO- or AUTO-HSCT, respectively (P=NS). Although the cumulative incidence (CI) of relapse was lower in ALLO- than in AUTO-HSCT recipients (17% vs 28%, respectively; P=0.043), the CI of TRM was 7% in both groups. Patients transplanted with unrelated donor cord blood had a remarkable 92.3% 8-year DFS probability. Altogether, these data confirm that HSCT is a suitable option for preventing leukemia recurrence in HR children with CR1 AML.
    Subject(s): Autografts ; Follow-Up Studies ; Leukemia, Myeloid, Acute - pathology ; Humans ; Child, Preschool ; Hematopoietic Stem Cell Transplantation ; Infant ; Male ; Survival Rate ; Abnormal Karyotype ; Cord Blood Stem Cell Transplantation ; Leukemia, Myeloid, Acute - mortality ; fms-Like Tyrosine Kinase 3 - genetics ; Disease-Free Survival ; Allografts ; Adolescent ; Myeloablative Agonists - administration & dosage ; Female ; Transplantation Conditioning - methods ; Child ; Leukemia, Myeloid, Acute - therapy ; Leukemia, Myeloid, Acute - genetics ; Transplantation ; Health aspects ; Patient outcomes ; Hematopoietic stem cells ; Index Medicus
    ISSN: 0268-3369
    E-ISSN: 1476-5365
    Source: Nature Open Access
    Source: Academic Search Ultimate
    Source: Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals
    Source: Alma/SFX Local Collection
    Source: © ProQuest LLC All rights reserved〈img src="https://exlibris-pub.s3.amazonaws.com/PQ_Logo.jpg" style="vertical-align:middle;margin-left:7px"〉
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  • 7
    Language: English
    In: Leukemia, 2012, Vol.26 (7), p.1681-1684
    Subject(s): Hematologic and hematopoietic diseases ; Biological and medical sciences ; Medical sciences ; Prognosis ; Receptors, Tumor Necrosis Factor, Type I - blood ; Enzyme-Linked Immunosorbent Assay ; Interleukin-2 Receptor alpha Subunit - blood ; Humans ; Monitoring, Immunologic ; Child, Preschool ; Male ; Graft vs Host Disease - blood ; Elafin - blood ; Graft vs Host Disease - immunology ; Young Adult ; Mesenchymal Stromal Cells - cytology ; Bone Marrow - metabolism ; Adolescent ; Adult ; Female ; Graft vs Host Disease - prevention & control ; Child ; Mesenchymal Stem Cell Transplantation ; Care and treatment ; Immunology ; Stem cells ; Graft versus host reaction ; Transplantation ; Diagnosis ; Research ; Methods ; Index Medicus
    ISSN: 0887-6924
    E-ISSN: 1476-5551
    Source: Nature Open Access
    Source: Academic Search Ultimate
    Source: Alma/SFX Local Collection
    Source: © ProQuest LLC All rights reserved〈img src="https://exlibris-pub.s3.amazonaws.com/PQ_Logo.jpg" style="vertical-align:middle;margin-left:7px"〉
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  • 8
    Language: English
    In: Bone marrow transplantation (Basingstoke), 2007-08, Vol.40 (3), p.225-233
    Description: Hurler's syndrome (HS), the most severe form of mucopolysaccharidosis type-I, causes progressive deterioration of the central nervous system and death in childhood. Allogeneic stem cell transplantation (SCT) before the age of 2 years halts disease progression. Graft failure limits the success of SCT. We analyzed data on HS patients transplanted in Europe to identify the risk factors for graft failure. We compared outcomes in 146 HS patients transplanted with various conditioning regimens and grafts. Patients were transplanted between 1994 and 2004 and registered to the European Blood and Marrow Transplantation database. Risk factor analysis was performed using logistic regression. 'Survival' and 'alive and engrafted'-rate after first SCT was 85 and 56%, respectively. In multivariable analysis, T-cell depletion (odds ratio (OR) 0.18; 95% confidence interval (CI) 0.04-0.71; P=0.02) and reduced-intensity conditioning (OR 0.08; 95% CI 0.02-0.39; P=0.002) were the risk factors for graft failure. Busulfan targeting protected against graft failure (OR 5.76; 95% CI 1.20-27.54; P=0.028). No difference was noted between cell sources used (bone marrow, peripheral blood stem cells or cord blood (CB)); however, significantly more patients who received CB transplants had full-donor chimerism (OR 9.31; 95% CI 1.06-82.03; P=0.044). These outcomes may impact the safety/efficacy of SCT for 'inborn-errors of metabolism' at large. CB increased the likelihood of sustained engraftment associated with normal enzyme levels and could therefore be considered as a preferential cell source in SCT for 'inborn errors of metabolism'.
    Subject(s): Errors of metabolism ; Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy ; Bone marrow, stem cells transplantation. Graft versus host reaction ; Transfusions. Complications. Transfusion reactions. Cell and gene therapy ; Biological and medical sciences ; Metabolic diseases ; Medical sciences ; Carbohydrates (enzymatic deficiencies). Glycogenosis ; Humans ; Child, Preschool ; Infant ; Male ; Busulfan - administration & dosage ; Transplantation, Homologous ; Graft Rejection - mortality ; Mucopolysaccharidosis I - therapy ; Female ; Transplantation Conditioning - adverse effects ; Retrospective Studies ; Child ; Infant, Newborn ; Databases, Factual ; Europe ; Risk Factors ; Hematopoietic Stem Cell Transplantation ; Graft Rejection - etiology ; Logistic Models ; Survival Rate ; Lymphocyte Depletion - adverse effects ; Transplantation Chimera ; Disease-Free Survival ; Mucopolysaccharidosis I - mortality ; Myeloablative Agonists - administration & dosage ; Graft Survival - drug effects ; Care and treatment ; Transplantation ; Hurler's syndrome ; Patient outcomes ; Hematopoietic stem cells ; Index Medicus ; inherited disorders ; cord blood
    ISSN: 0268-3369
    E-ISSN: 1476-5365
    Source: Nature Open Access
    Source: Academic Search Ultimate
    Source: Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals
    Source: Alma/SFX Local Collection
    Source: © ProQuest LLC All rights reserved〈img src="https://exlibris-pub.s3.amazonaws.com/PQ_Logo.jpg" style="vertical-align:middle;margin-left:7px"〉
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  • 9
    Language: English
    In: Journal of Geophysical Research: Solid Earth, 2010-11, Vol.115 (B11), p.n/a
    Description: Fracture reactivation is a widespread process in nature even though evidence of magma‐induced reactivation is less documented. Here we provide evidence of the reactivation of a fracture system on the upper flank of the Mt. Etna volcano and consider its possible implications in understanding the recent volcanic and tectonic activity. A NNW–SSE trending fracture, partly accompanied by magma emplacement in the form of a laterally propagating dike, formed in 1989 on the upper SE flank of Etna. Lava effusions in 1991–1993, 2001, and 2006 were associated with volcano‐tectonic (VT) seismicity and ground deformations on the upper part of the volcano, which document the seismogenetic involvement of the 1989 fractures, although without eruptive phenomena along the discontinuity. In addition to the aforementioned episodes of VT seismicity, differences in the characteristics of the background seismic radiation (volcanic tremor) were measured at stations close to these fractures during the eruptive activity on 24 November 2006, for which more detailed volcanological and seismological time histories are available. Moving on from these findings, we analyze volcanic tremor data recorded close to the summit and along the S flank of the volcano to highlight the interactions between seismic radiation and the 1989 fracture system. Centroid location of volcanic tremor and wave field characteristics at stations of the permanent local seismic network of Etna highlight the guidance role played by the 1989 fractures during the eruptive activity on 24 November 2006. In addition, the collected data shed light on hitherto unknown structural features, which appear to connect the volcano summit to the lower SE slope and also play an important role in controlling the instability of the E flank. More generally, this study shows how (1) using an integrated approach, it is possible to link apparently different features to a common structure, showing uniform and distinct dynamics relevant at the volcano scale, and (2) fracture reactivation can also occur by means of magma intrusion, playing an important role in the transfer of magma within a volcanic edifice.
    Subject(s): volcanic tremor ; fracture reactivation ; seismic data processing ; Mt. Etna ; multidisciplinary study ; volcano‐tectonics ; Earth, ocean, space ; Earth sciences ; Exact sciences and technology
    ISSN: 0148-0227
    E-ISSN: 2156-2202
    Source: Wiley Online Library All Backfiles
    Source: Alma/SFX Local Collection
    Source: EBSCOhost EJS
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  • 10
    Language: English
    In: Journal of Geophysical Research - Solid Earth, 2009-10-10, Vol.114 (B10), p.B10308-n/a
    Description: In this paper we investigate ground motion properties in the western part of the Pernicana fault. This is the major fault of Mount Etna and drives the dynamic evolution of the area. In a previous work, Rigano et al. (2008) showed that a significant horizontal polarization characterizes ground motion in fault zones of Mount Etna, both during earthquakes and ambient vibrations. We have performed denser microtremor measurements in the NE rift segment and in intensely deformed zones of the Pernicana fault at Piano Pernicana. This study includes mapping of azimuth‐dependent horizontal‐to‐vertical spectral ratios along and across the fault, frequency–wave number techniques applied to array data to investigate the nature of ambient vibrations, and polarization analysis through the conventional covariance matrix method. Our results indicate that microtremors are likely composed of volcanic tremor. Spectral ratios show strong directional resonances of horizontal components around 1 Hz when measurements enter the most damaged part of the fault zone. Their polarization directions show an abrupt change, by 20° to 40°, at close measurements between the northern and southern part of the fault zone. Recordings of local earthquakes at one site in the fault zone confirm the occurrence of polarization with the same angle found using volcanic tremor. We have also found that the directional effect is not time‐dependent, at least at a seasonal scale. This observation and the similar behavior of volcanic tremors and earthquake‐induced ground motions suggest that horizontal polarization is the effect of local fault properties. However, the 1‐Hz resonant frequency cannot be reproduced using the 1‐D vertically varying model inferred from the array data analysis, suggesting a role of lateral variations of the fault zone. Although the actual cause of polarization is unknown, a role of stress‐induced anisotropy and microfracture orientation in the near‐surface lavas of the Pernicana fault can be hypothesized consistently with the sharp rotation of the polarization angle within the damaged fault zone.
    Subject(s): High strain deformation zones ; Structural Geology ; Fractures and faults ; Seismology ; Volcano seismology ; Earthquake ground motions and engineering seismology ; volcanic tremor ; directional resonance ; polarization ; fault zone ; Pernicana fault
    ISSN: 0148-0227
    E-ISSN: 2156-2202
    Source: Wiley Online Library All Backfiles
    Source: Alma/SFX Local Collection
    Source: EBSCOhost EJS
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