Neonatal gene transfer using lentiviral vector for murine Pompe disease: long-term expression and glycogen reduction.
about
Targeted approaches to induce immune tolerance for Pompe disease therapyRH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy.Neonatal helper-dependent adenoviral vector gene therapy mediates correction of hemophilia A and tolerance to human factor VIIIGene therapy approaches for lysosomal storage disorders, a good model for the treatment of mendelian diseases.Disease modeling and lentiviral gene transfer in patient-specific induced pluripotent stem cells from late-onset Pompe disease patient.Altering α-dystroglycan receptor affinity of LCMV pseudotyped lentivirus yields unique cell and tissue tropismAdministration of anti-CD3 antibodies modulates the immune response to an infusion of α-glucosidase in miceTFEB overexpression promotes glycogen clearance of Pompe disease iPSC-derived skeletal muscle.Minimum requirement of donor cells to reduce the glycolipid storage following bone marrow transplantation in a murine model of Fabry disease.The humanistic burden of Pompe disease: are there still unmet needs? A systematic review.
P2860
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P2860
Neonatal gene transfer using lentiviral vector for murine Pompe disease: long-term expression and glycogen reduction.
description
2009 nî lūn-bûn
@nan
2009年の論文
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2009年学术文章
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2009年学术文章
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2009年学术文章
@zh-cn
2009年学术文章
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2009年学术文章
@zh-my
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2009年學術文章
@zh-hant
name
Neonatal gene transfer using l ...... ession and glycogen reduction.
@en
Neonatal gene transfer using l ...... ession and glycogen reduction.
@nl
type
label
Neonatal gene transfer using l ...... ession and glycogen reduction.
@en
Neonatal gene transfer using l ...... ession and glycogen reduction.
@nl
prefLabel
Neonatal gene transfer using l ...... ession and glycogen reduction.
@en
Neonatal gene transfer using l ...... ession and glycogen reduction.
@nl
P2093
P2860
P356
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Neonatal gene transfer using l ...... ession and glycogen reduction.
@en
P2093
H Kobayashi
S O Kyosen
P2860
P2888
P304
P356
10.1038/GT.2009.160
P577
2009-12-24T00:00:00Z
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P6179
1030322456