Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome.
about
Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice.Retroviral-vector-mediated gene therapy to mucopolysaccharidosis I mice improves sensorimotor impairments and other behavioral deficits.Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.Cell- and gene-based therapeutic approaches for neurological deficits in mucopolysaccharidosesSecreted luciferase for in vivo evaluation of systemic protein delivery in miceMulti-system disorders of glycosphingolipid and ganglioside metabolismTherapeutic approaches for lysosomal storage diseasesHematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome)K-Cl cotransporter gene expression during human and murine erythroid differentiation.Gene therapy for neurologic manifestations of mucopolysaccharidoses.Normalization and improvement of CNS deficits in mice with Hurler syndrome after long-term peripheral delivery of BBB-targeted iduronidase.Progression of Behavioral and CNS Deficits in a Viable Murine Model of Chronic Neuronopathic Gaucher Disease.Gene therapy approaches for lysosomal storage disease: next-generation treatment.Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?Engineering a lysosomal enzyme with a derivative of receptor-binding domain of apoE enables delivery across the blood-brain barrierFeatures of brain MRI in dogs with treated and untreated mucopolysaccharidosis type IPlatelets are efficient and protective depots for storage, distribution, and delivery of lysosomal enzyme in mice with Hurler syndrome.Recent advances in lentiviral vector development and applications.Gene therapy for the neurological manifestations in lysosomal storage disorders.Liver-Directed Human Amniotic Epithelial Cell Transplantation Improves Systemic Disease Phenotype in Hurler Syndrome Mouse Model.Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications.Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I.High and prolonged sulfamidase secretion by the liver of MPS-IIIA mice following hydrodynamic tail vein delivery of antibiotic-free pFAR4 plasmid vector.Comprehensive evaluation of blood-brain barrier-forming micro-vasculatures: Reference and marker genes with cellular composition.Getting the Most: Enhancing Efficacy by Promoting Erythropoiesis and Thrombopoiesis after Gene Therapy in Mice with Hurler Syndrome
P2860
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P2860
Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome.
description
2009 nî lūn-bûn
@nan
2009年の論文
@ja
2009年学术文章
@wuu
2009年学术文章
@zh-cn
2009年学术文章
@zh-hans
2009年学术文章
@zh-my
2009年学术文章
@zh-sg
2009年學術文章
@yue
2009年學術文章
@zh
2009年學術文章
@zh-hant
name
Reprogramming erythroid cells ...... in mice with Hurler syndrome.
@en
Reprogramming erythroid cells ...... in mice with Hurler syndrome.
@nl
type
label
Reprogramming erythroid cells ...... in mice with Hurler syndrome.
@en
Reprogramming erythroid cells ...... in mice with Hurler syndrome.
@nl
prefLabel
Reprogramming erythroid cells ...... in mice with Hurler syndrome.
@en
Reprogramming erythroid cells ...... in mice with Hurler syndrome.
@nl
P2093
P2860
P356
P1476
Reprogramming erythroid cells ...... in mice with Hurler syndrome.
@en
P2093
Daren Wang
Gregory Grabowski
Punam Malik
Stella Davies
P2860
P304
19958-19963
P356
10.1073/PNAS.0908528106
P407
P577
2009-11-10T00:00:00Z