Retroviral vector design studies toward hematopoietic stem cell gene therapy for mucopolysaccharidosis type I.
about
Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidosesDirect gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I.Normalization and improvement of CNS deficits in mice with Hurler syndrome after long-term peripheral delivery of BBB-targeted iduronidase.Engineering a lysosomal enzyme with a derivative of receptor-binding domain of apoE enables delivery across the blood-brain barrierReprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome.Co-expression of MGMT(P140K) and alpha-L-iduronidase in primary hepatocytes from mucopolysaccharidosis type I mice enables efficient selection with metabolic correction.Elements of lentiviral vector design toward gene therapy for treating mucopolysaccharidosis I.Treatment of adult MPSI mouse brains with IDUA-expressing mesenchymal stem cells decreases GAG deposition and improves exploratory behavior.In vitro gene therapy of mucopolysaccharidosis type I by lentiviral vectors.
P2860
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P2860
Retroviral vector design studies toward hematopoietic stem cell gene therapy for mucopolysaccharidosis type I.
description
2000 nî lūn-bûn
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2000年の論文
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2000年学术文章
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2000年学术文章
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2000年学术文章
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2000年学术文章
@zh-my
2000年学术文章
@zh-sg
2000年學術文章
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2000年學術文章
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2000年學術文章
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name
Retroviral vector design studi ...... mucopolysaccharidosis type I.
@en
type
label
Retroviral vector design studi ...... mucopolysaccharidosis type I.
@en
prefLabel
Retroviral vector design studi ...... mucopolysaccharidosis type I.
@en
P2093
P2860
P356
P1433
P1476
Retroviral vector design studi ...... mucopolysaccharidosis type I.
@en
P2093
P2860
P2888
P304
P356
10.1038/SJ.GT.3301298
P577
2000-11-01T00:00:00Z
P5875
P6179
1027725104