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1. Full Text Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
by Alessandra Biffi and Eugenio Montini and Laura Lorioli and Martina Cesani and Francesca Fumagalli and Tiziana Plati and Cristina Baldoli and Sabata Martino and Andrea Calabria and Sabrina Canale and Fabrizio Benedicenti and Giuliana Vallanti and Luca Biasco and Simone Leo and Nabil Kabbara and Gianluigi Zanetti and William B. Rizzo and Nalini A. L. Mehta and Maria Pia Cicalese and Miriam Casiraghi and Jaap J. Boelens and Ubaldo Del Carro and David J. Dow and Manfred Schmidt and Andrea Assanelli and Victor Neduva and Clelia Di Serio and Elia Stupka and Jason Gardner and Christof von Kalle and Claudio Bordignon and Fabio Ciceri and Attilio Rovelli and Maria Grazia Roncarolo and Alessandro Aiuti and Maria Sessa and Luigi Naldini
Science (American Association for the Advancement of Science), ISSN 0036-8075, 8/2013, Volume 341, Issue 6148, pp. 864 - 864
RESEARCH ARTICLE SUMMARY | Metachromatic leukodystrophy | Nervous system diseases | Neurodegenerative diseases | Demyelinating diseases | Medical treatment | Gene therapy | Hematopoietic stem cells | Image contrast | Science & Technology - Other Topics | Multidisciplinary Sciences | Science & Technology | Transduction, Genetic | Follow-Up Studies | Humans | Hematopoietic Stem Cell Transplantation | Leukodystrophy, Metachromatic - therapy | Treatment Outcome | Hematopoietic Stem Cells - metabolism | Virus Integration | Lentivirus | Magnetic Resonance Imaging | Genetic Engineering | Brain - pathology | Genetic Vectors - toxicity | DNA Damage | Leukodystrophy, Metachromatic - pathology | Cerebroside-Sulfatase - genetics | Genetic Therapy - methods | Index Medicus
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2. Full Text Mutation Update of ARSA and PSAP Genes Causing Metachromatic Leukodystrophy
by Cesani, Martina and Lorioli, Laura and Grossi, Serena and Amico, Giulia and Fumagalli, Francesca and Spiga, Ivana and Filocamo, Mirella and Biffi, Alessandra
Human mutation, ISSN 1059-7794, 01/2016, Volume 37, Issue 1, pp. 16 - 27
metachromatic leukodystrophy | ARSA | mutation | arylsulfatase A | saposin B | PSAP | Metachromatic leukodystrophy | Arylsulfatase A | Mutation | Saposin B | Life Sciences & Biomedicine | Genetics & Heredity | Science & Technology | Leukodystrophy, Metachromatic - genetics | Phenotype | Leukodystrophy, Metachromatic - diagnosis | Genetic Association Studies | Humans | Alleles | Databases, Genetic | Saposins - genetics | Genotype | Cerebroside-Sulfatase - genetics | Sphingolipidoses | Enzymes | Nervous system diseases | Genetic aspects | Genes | Proteins | Genetic disorders | Cells | Index Medicus
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3. Full Text Metallothioneins are neuroprotective agents in lysosomal storage disorders
by Cavalca, Eleonora and Cesani, Martina and Gifford, Jennifer C and Sena-Esteves, Miguel and Terreni, Maria Rosa and Leoncini, Giuseppe and Peviani, Marco and Biffi, Alessandra
Annals of neurology, ISSN 0364-5134, 02/2018, Volume 83, Issue 2, pp. 418 - 432
Neurosciences | Clinical Neurology | Neurosciences & Neurology | Life Sciences & Biomedicine | Science & Technology | Gene Transfer Techniques | Neuroprotective Agents | Animals | Humans | Mice, Inbred C57BL | Mice, Transgenic | Mice | Lysosomal Storage Diseases - pathology | Metallothionein | Neuroprotection | Oxidative stress | Animal models | Disease | Central nervous system | Disorders | Lysosomal storage diseases | Neuroprotective agents | Proteins | Purkinje cells | Lysosomal enzymes | Rodents | Degeneration | Phenotypes | Gene transfer | Transgenic mice | Pharmacology | Gene expression | Chemical compounds | Survival | Microglia | Storage | Metallothioneins | Metabolic disorders | Apoptosis | Index Medicus
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4. Full Text Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection
by Biffi, Alessandra and Bartholomae, Cynthia C and Cesana, Daniela and Cartier, Natalie and Aubourg, Patrik and Ranzani, Marco and Cesani, Martina and Benedicenti, Fabrizio and Plati, Tiziana and Rubagotti, Enrico and Merella, Stefania and Capotondo, Alessia and Sgualdino, Jacopo and Zanetti, Gianluigi and Von Kalle, Christof and Schmidt, Manfred and Naldini, Luigi and Montini, Eugenio
Blood, ISSN 0006-4971, 05/2011, Volume 117, Issue 20, pp. 5332 - 5339
Life Sciences & Biomedicine | Hematology | Science & Technology | Hematologic and hematopoietic diseases | Biological and medical sciences | Medical sciences | Gene Transfer Techniques | Virus Integration - genetics | Humans | Hematopoietic Stem Cell Transplantation | Clinical Trials as Topic | DNA-Binding Proteins - genetics | Interleukin Receptor Common gamma Subunit - genetics | DNA-Binding Proteins - deficiency | Mice, Knockout | Transplantation Chimera - genetics | Interleukin Receptor Common gamma Subunit - deficiency | Animals | Adrenoleukodystrophy - therapy | Lentivirus - genetics | Mice | Adrenoleukodystrophy - genetics | Genetic Vectors | Genetic Therapy - methods | Index Medicus | Abridged Index Medicus
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5. Full Text Metallothioneins are neuroprotective agents in lysosomal storage disorders
: Metallothioneins as Neuroprotective Agents in LSDs
by Cavalca, Eleonora and Cesani, Martina and Gifford, Jennifer C and Sena-Esteves, Miguel and Terreni, Maria Rosa and Leoncini, Giuseppe and Peviani, Marco and Biffi, Alessandra
Annals of neurology, ISSN 0364-5134, 02/2018, Volume 83, Issue 2, pp. 418 - 432
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6. Full Text Metallothioneins as dynamic markers for brain disease in lysosomal disorders
by Cesani, Martina and Cavalca, Eleonora and Macco, Romina and Leoncini, Giuseppe and Terreni, Maria Rosa and Lorioli, Laura and Furlan, Roberto and Comi, Giancarlo and Doglioni, Claudio and Zacchetti, Daniele and Sessa, Maria and Scherzer, Clemens R and Biffi, Alessandra
Annals of neurology, ISSN 0364-5134, 01/2014, Volume 75, Issue 1, pp. 127 - 137
Neurosciences | Clinical Neurology | Neurosciences & Neurology | Life Sciences & Biomedicine | Science & Technology | Disease | Patients | Rodents | Index Medicus | Original
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7. Human hematopoietic stem cells in gene therapy: Pre-clinical and clinical issues
by Biffi, Alessandra and Cesani, Martina
Current gene therapy, ISSN 1566-5232, 04/2008, Volume 8, Issue 2, pp. 135 - 146
Transduction | Ex vivo manipulation | Gene therapy | Hematopoietic stem cells | Life Sciences & Biomedicine | Genetics & Heredity | Science & Technology | Viruses - genetics | Hematopoietic Stem Cells - cytology | Humans | Hematopoietic Stem Cells - metabolism | Transduction, Genetic - methods | Genetic Vectors - genetics | Genetic Therapy - methods
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8. Full Text Mutation Update ofARSAandPSAPGenes Causing Metachromatic Leukodystrophy
by Cesani, Martina and Lorioli, Laura and Grossi, Serena and Amico, Giulia and Fumagalli, Francesca and Spiga, Ivana and Filocamo, Mirella and Biffi, Alessandra
Human mutation, ISSN 1059-7794, 01/2016, Volume 37, Issue 1, pp. 16 - 27
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9. Full Text Critical issues for the proper diagnosis of Metachromatic Leukodystrophy
by Lorioli, Laura and Cesani, Martina and Regis, Stefano and Morena, Francesco and Grossi, Serena and Fumagalli, Francesca and Acquati, Serena and Redaelli, Daniela and Pini, Antonella and Sessa, Maria and Martino, Sabata and Filocamo, Mirella and Biffi, Alessandra
Gene, ISSN 0378-1119, 03/2014, Volume 537, Issue 2, pp. 348 - 351
Arylsulfatase A | Metachromatic Leukodystrophy | Molecular diagnosis | Enzymatic activity | Life Sciences & Biomedicine | Genetics & Heredity | Science & Technology | Leukodystrophy, Metachromatic - genetics | Leukodystrophy, Metachromatic - diagnosis | Humans | Alleles | Female | Heterozygote | Infant | Male | Cerebroside-Sulfatase - blood | Cerebroside-Sulfatase - genetics | Sphingolipidoses | Enzymes | Diagnosis | Analysis | Index Medicus
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