Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice.
about
Ataxia is the major neuropathological finding in arylsulfatase G-deficient mice: similarities and dissimilarities to Sanfilippo disease (mucopolysaccharidosis type III).Gene therapy for deafness.Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III.Therapies of mucopolysaccharidosis IVA (Morquio A syndrome).Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?Mucopolysaccharidosis IIIB confers enhanced neonatal intracranial transduction by AAV8 but not by 5, 9 or rh10Hematopoietic Stem cell transplantation and lentiviral vector-based gene therapy for Krabbe's disease: Present convictions and future prospects.Reversibility of neuropathology in Tay-Sachs-related diseases.Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.Preferred transduction with AAV8 and AAV9 via thalamic administration in the MPS IIIB model: A comparison of four rAAV serotypes.miRNA-mediated post-transcriptional silencing of transgenes leads to increased adeno-associated viral vector yield and targeting specificity.Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice.Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy.How close are we to therapies for Sanfilippo disease?Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy.Sanfilippo syndrome: Overall reviewA model of mucopolysaccharidosis type IIIB in pigs
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P2860
Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice.
description
2013 nî lūn-bûn
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2013年の論文
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2013年学术文章
@wuu
2013年学术文章
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2013年学术文章
@zh-cn
2013年学术文章
@zh-hans
2013年学术文章
@zh-my
2013年学术文章
@zh-sg
2013年學術文章
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2013年學術文章
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name
Disease correction by combined ...... nfilippo Syndrome type B mice.
@en
Disease correction by combined ...... nfilippo Syndrome type B mice.
@nl
type
label
Disease correction by combined ...... nfilippo Syndrome type B mice.
@en
Disease correction by combined ...... nfilippo Syndrome type B mice.
@nl
prefLabel
Disease correction by combined ...... nfilippo Syndrome type B mice.
@en
Disease correction by combined ...... nfilippo Syndrome type B mice.
@nl
P2093
P2860
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Disease correction by combined ...... anfilippo Syndrome type B mice
@en
P2093
B E Crawford
J L Orrock
K K Ohlemiller
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P2888
P304
P356
10.1038/GT.2013.14
P577
2013-03-28T00:00:00Z
P5875
P6179
1026985145