Gene therapy approaches for lysosomal storage disorders, a good model for the treatment of mendelian diseases
出版年份 2012 全文链接
标题
Gene therapy approaches for lysosomal storage disorders, a good model for the treatment of mendelian diseases
作者
关键词
-
出版物
ACTA PAEDIATRICA
Volume 101, Issue 7, Pages 692-701
出版商
Wiley
发表日期
2012-03-17
DOI
10.1111/j.1651-2227.2012.02674.x
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。- Gene Therapy for Lysosomal Storage Diseases (LSDs) in Large Animal Models
- (2013) M. Haskins ILAR JOURNAL
- Hybrids of Nonviral Vectors for Gene Delivery
- (2010) Shubiao Zhang et al. BIOCONJUGATE CHEMISTRY
- Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model
- (2010) I. Visigalli et al. BLOOD
- Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency
- (2010) Francesca Ferrua et al. Current Opinion in Allergy and Clinical Immunology
- Gene delivery by lipoplexes and polyplexes
- (2010) Conchita Tros de Ilarduya et al. EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES
- Restoration of central nervous system α-N-acetylglucosaminidase activity and therapeutic benefits in mucopolysaccharidosis IIIB mice by a single intracisternal recombinant adeno-associated viral type 2 vector delivery
- (2010) Haiyan Fu et al. JOURNAL OF GENE MEDICINE
- The next step in gene delivery: Molecular engineering of adeno-associated virus serotypes
- (2010) Jinhui Wang et al. JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY
- AAV-Mediated Gene Delivery in Adult GM1-Gangliosidosis Mice Corrects Lysosomal Storage in CNS and Improves Survival
- (2010) Rena C. Baek et al. PLoS One
- Nonviral Gene Delivery: Principle, Limitations, and Recent Progress
- (2009) Mohammed S. Al-Dosari et al. AAPS Journal
- Widespread biochemical correction of murine mucopolysaccharidosis type VII pathology by liver hydrodynamic plasmid delivery
- (2009) M Richard et al. GENE THERAPY
- Neonatal gene transfer using lentiviral vector for murine Pompe disease: long-term expression and glycogen reduction
- (2009) S O Kyosen et al. GENE THERAPY
- The trigeminal retrograde transfer pathway in the treatment of neurodegeneration
- (2009) Stephanos Kyrkanides et al. JOURNAL OF NEUROIMMUNOLOGY
- GAP43 overexpression and enhanced neurite outgrowth in mucopolysaccharidosis type IIIB cortical neuron cultures
- (2009) Michaël Hocquemiller et al. JOURNAL OF NEUROSCIENCE RESEARCH
- Lentiviral-mediated correction of MPS VI cells and gene transfer to joint tissues
- (2009) Sharon Byers et al. MOLECULAR GENETICS AND METABOLISM
- Systemic Correction of Storage Disease in MPS I NOD/SCID Mice Using the Sleeping Beauty Transposon System
- (2009) Elena L Aronovich et al. MOLECULAR THERAPY
- CNS-directed gene therapy for lysosomal storage diseases
- (2008) Mark S Sands et al. ACTA PAEDIATRICA
- Metachromatic leukodystrophy: genetics, pathogenesis and therapeutic options
- (2008) Volkmar Gieselmann ACTA PAEDIATRICA
- Gene therapy of Hunter syndrome: Evaluation of the efficiency of muscle electro gene transfer for the production and release of recombinant iduronate-2-sulfatase (IDS)
- (2008) A. Friso et al. BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE
- Lysosomal disorders: From storage to cellular damage
- (2008) Andrea Ballabio et al. BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH
- Intranasal delivery bypasses the blood-brain barrier to target therapeutic agents to the central nervous system and treat neurodegenerative disease
- (2008) Leah R Hanson et al. BMC NEUROSCIENCE
- Lysosomal Storage Diseases and the Blood-Brain Barrier
- (2008) David Begley et al. CURRENT PHARMACEUTICAL DESIGN
- Nuclear-targeted chimeric vector enhancing nonviral gene transfer into skeletal muscle of Fabry mice in vivo
- (2008) Matthieu D. Lavigne et al. FASEB JOURNAL
- 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
- Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
- (2008) Steven J. Howe et al. JOURNAL OF CLINICAL INVESTIGATION
- Nonviral gene transfection nanoparticles: function and applications in the brain
- (2008) Indrajit Roy et al. Nanomedicine-Nanotechnology Biology and Medicine
- Central nervous system therapy for lysosomal storage disorders
- (2008) Gregory M. Enns et al. Neurosurgical Focus
- Biochemical, Pathological, and Skeletal Improvement of Mucopolysaccharidosis VI After Gene Transfer to Liver but Not to Muscle
- (2007) Alessandra Tessitore et al. MOLECULAR THERAPY
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