Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy
出版年份 2014 全文链接
标题
Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy
作者
关键词
-
出版物
HUMAN MOLECULAR GENETICS
Volume 23, Issue 12, Pages 3250-3268
出版商
Oxford University Press (OUP)
发表日期
2014-01-25
DOI
10.1093/hmg/ddu034
参考文献
相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。- Region- and Age-Dependent Alterations of Glial-Neuronal Metabolic Interactions Correlate with CNS Pathology in a Mouse Model of Globoid Cell Leukodystrophy
- (2013) Tore Wergeland Meisingset et al. JOURNAL OF CEREBRAL BLOOD FLOW AND METABOLISM
- Therapeutic Response in Feline Sandhoff Disease Despite Immunity to Intracranial Gene Therapy
- (2013) Allison M Bradbury et al. MOLECULAR THERAPY
- Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome
- (2013) A. Aiuti et al. SCIENCE
- Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
- (2013) A. Biffi et al. SCIENCE
- Multipotent Stromal Cells Alleviate Inflammation, Neuropathology, and Symptoms Associated with Globoid Cell Leukodystrophy in the Twitcher Mouse
- (2013) Brittni A. Scruggs et al. STEM CELLS
- Correction of Brain Oligodendrocytes by AAVrh.10 Intracerebral Gene Therapy in Metachromatic Leukodystrophy Mice
- (2012) Françoise Piguet et al. HUMAN GENE THERAPY
- Correction of Pathological Accumulation of Glycosaminoglycans in Central Nervous System and Peripheral Tissues of MPSIIIA Mice Through Systemic AAV9 Gene Transfer
- (2012) Albert Ruzo et al. HUMAN GENE THERAPY
- The galactocerebrosidase enzyme contributes to maintain a functional neurogenic niche during early post-natal CNS development
- (2012) Sara Santambrogio et al. HUMAN MOLECULAR GENETICS
- Bone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine model of globoid cell leukodystrophy
- (2012) Elizabeth Y. Qin et al. MOLECULAR GENETICS AND METABOLISM
- Extended Normal Life After AAVrh10-mediated Gene Therapy in the Mouse Model of Krabbe Disease
- (2012) Mohammad A Rafi et al. MOLECULAR THERAPY
- Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation
- (2012) A. Capotondo et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Axonopathy is a compounding factor in the pathogenesis of Krabbe disease
- (2011) Ludovico Cantuti Castelvetri et al. ACTA NEUROPATHOLOGICA
- Peripheral Neuropathy in the Twitcher Mouse Involves the Activation of Axonal Caspase 3
- (2011) Benjamin Smith et al. ASN Neuro
- Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection
- (2011) A. Biffi et al. BLOOD
- Programmed cell death during postnatal development of the rodent nervous system
- (2011) Woon Ryoung Kim et al. DEVELOPMENT GROWTH & DIFFERENTIATION
- Adult neurogenesis in mammals - a theme with many variations
- (2011) Luca Bonfanti et al. EUROPEAN JOURNAL OF NEUROSCIENCE
- Gene therapy for lysosomal storage disorders
- (2011) Angela Gritti EXPERT OPINION ON BIOLOGICAL THERAPY
- Corticospinal tract transduction: a comparison of seven adeno-associated viral vector serotypes and a non-integrating lentiviral vector
- (2011) T H Hutson et al. GENE THERAPY
- Bone Marrow Transplantation Augments the Effect of Brain- and Spinal Cord-Directed Adeno-Associated Virus 2/5 Gene Therapy by Altering Inflammation in the Murine Model of Globoid-Cell Leukodystrophy
- (2011) A. S. Reddy et al. JOURNAL OF NEUROSCIENCE
- Lentiviral Vector Integration Profiles Differ in Rodent Postmitotic Tissues
- (2011) Cynthia C Bartholomae et al. MOLECULAR THERAPY
- Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
- (2011) Federico Mingozzi et al. NATURE REVIEWS GENETICS
- Neural Stem Cell Gene Therapy Ameliorates Pathology and Function in a Mouse Model of Globoid Cell Leukodystrophy
- (2011) Margherita Neri et al. STEM CELLS
- A High-Resolution Anatomical Atlas of the Transcriptome in the Mouse Embryo
- (2011) Graciana Diez-Roux et al. PLOS BIOLOGY
- Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models
- (2010) Annalisa Lattanzi et al. HUMAN MOLECULAR GENETICS
- Genome-wide high-throughput integrome analyses by nrLAM-PCR and next-generation sequencing
- (2010) Anna Paruzynski et al. Nature Protocols
- Diffusion Tensor Imaging Detects Abnormalities in the Corticospinal Tracts of Neonates with Infantile Krabbe Disease
- (2009) M.L. Escolar et al. AMERICAN JOURNAL OF NEURORADIOLOGY
- Specific Determination of -Galactocerebrosidase Activity via Competitive Inhibition of -Galactosidase
- (2009) S. Martino et al. CLINICAL CHEMISTRY
- The long-term outcomes of presymptomatic infants transplanted for Krabbe disease: Report of the workshop held on July 11 and 12, 2008, Holiday Valley, New York
- (2009) Patricia K Duffner et al. GENETICS IN MEDICINE
- Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate
- (2009) M.-A. Colle et al. HUMAN MOLECULAR GENETICS
- Combined hematopoietic and lentiviral gene-transfer therapies in newborn Twitcher mice reveal contemporaneous neurodegeneration and demyelination in Krabbe disease
- (2009) F. Galbiati et al. JOURNAL OF NEUROSCIENCE RESEARCH
- Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
- (2009) N. Cartier et al. SCIENCE
- Mechanisms of distribution of mouse β-galactosidase in the adult GM1-gangliosidosis brain
- (2008) M L D Broekman et al. GENE THERAPY
- Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis by CNS Administration of a Serotype 2 Adeno-Associated Virus Expressing CLN2 cDNA
- (2008) Stefan Worgall et al. HUMAN GENE THERAPY
- Expanded Repertoire of AAV Vector Serotypes Mediate Unique Patterns of Transduction in Mouse Brain
- (2008) Cassia N Cearley et al. MOLECULAR THERAPY
Publish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn MoreBecome a Peeref-certified reviewer
The Peeref Institute provides free reviewer training that teaches the core competencies of the academic peer review process.
Get Started