Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I.
about
Therapies for the bone in mucopolysaccharidosesTranscranial pulsed ultrasound facilitates brain uptake of laronidase in enzyme replacement therapy for Mucopolysaccharidosis type I diseaseEvidence of a progressive motor dysfunction in Mucopolysaccharidosis type I mice.Retroviral-vector-mediated gene therapy to mucopolysaccharidosis I mice improves sensorimotor impairments and other behavioral deficits.Similar therapeutic efficacy between a single administration of gene therapy and multiple administrations of recombinant enzyme in a mouse model of lysosomal storage diseaseAssessment of dysmyelination with RAFFn MRI: application to murine MPS I.Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouseComparison of Endovascular and Intraventricular Gene Therapy With Adeno-Associated Virus-α-L-Iduronidase for Hurler DiseaseTherapies of mucopolysaccharidosis IVA (Morquio A syndrome).Lysosomal storage disease: gene therapy on both sides of the blood-brain barrier.Gene therapy for neurologic manifestations of mucopolysaccharidoses.Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis IWidespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna.Gene therapy approaches for lysosomal storage disease: next-generation treatment.Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?AAV Gene Therapy for MPS1-associated Corneal BlindnessLow-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease.Animal models for metabolic, neuromuscular and ophthalmological rare diseases.Standardization of α-L-iduronidase enzyme assay with Michaelis-Menten kinetics.Gene delivery strategies for the treatment of mucopolysaccharidoses.Proteomic analysis of mucopolysaccharidosis I mouse brain with two-dimensional polyacrylamide gel electrophoresis.Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.High-dose enzyme replacement therapy in murine Hurler syndromeIntranasal 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.Elements of lentiviral vector design toward gene therapy for treating mucopolysaccharidosis I.Correction of murine mucopolysaccharidosis type IIIA central nervous system pathology by intracerebroventricular lentiviral-mediated gene delivery.Increased longevity and metabolic correction following syngeneic BMT in a murine model of mucopolysaccharidosis type I.RTB lectin-mediated delivery of lysosomal α-l-iduronidase mitigates disease manifestations systemically including the central nervous system.Subregional brain distribution of simple and complex glycosphingolipids in the mucopolysaccharidosis type I (Hurler syndrome) mouse: impact of diet.Metabolic etiologies in West syndrome.
P2860
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P2860
Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I.
description
2011 nî lūn-bûn
@nan
2011年の論文
@ja
2011年論文
@yue
2011年論文
@zh-hant
2011年論文
@zh-hk
2011年論文
@zh-mo
2011年論文
@zh-tw
2011年论文
@wuu
2011年论文
@zh
2011年论文
@zh-cn
name
Direct gene transfer to the CN ...... mucopolysaccharidosis type I.
@ast
Direct gene transfer to the CN ...... mucopolysaccharidosis type I.
@en
type
label
Direct gene transfer to the CN ...... mucopolysaccharidosis type I.
@ast
Direct gene transfer to the CN ...... mucopolysaccharidosis type I.
@en
prefLabel
Direct gene transfer to the CN ...... mucopolysaccharidosis type I.
@ast
Direct gene transfer to the CN ...... mucopolysaccharidosis type I.
@en
P2093
P2860
P1476
Direct gene transfer to the CN ...... mucopolysaccharidosis type I.
@en
P2093
Andrew W Lenander
Chester B Whitley
Daniel A Wolf
Lalitha R Belur
Pankaj Gupta
R Scott McIvor
Walter C Low
Zhenhong Nan
P2860
P304
P356
10.1016/J.NBD.2011.02.015
P577
2011-03-17T00:00:00Z