A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice.

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A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice.

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2017 nî lūn-bûn

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2017年の論文

@ja

2017年論文

@yue

2017年論文

@zh-hant

2017年論文

@zh-hk

2017年論文

@zh-mo

2017年論文

@zh-tw

2017年论文

@wuu

2017年论文

@zh

2017年论文

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A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice.

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A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice.

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prefLabel

A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice.

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P1476

A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice

@en

P2093

Barry J Byrne

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Chia-Hao Cheng

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Darin J Falk

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Kai-Ling Chang

http://www.w3.org/2001/XMLSchema#string

Li-Kai Tsai

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Nien-Chu Shih

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Shin-Ichi Muramatsu

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P2860

Clinical and metabolic correction of pompe disease by enzyme therapy in acid maltase-deficient quail

Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease

Modulation of disease severity in mice with targeted disruption of the acid alpha-glucosidase gene

Targeted disruption of the acid alpha-glucosidase gene in mice causes an illness with critical features of both infantile and adult human glycogen storage disease type II

Retrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9.

The pharmacological chaperone AT2220 increases recombinant human acid α-glucosidase uptake and glycogen reduction in a mouse model of Pompe disease.

Peripheral nerve and neuromuscular junction pathology in Pompe disease

Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle

Pompe disease in infants: improving the prognosis by newborn screening and early treatment.

Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease

P2888

s12035-017-0763-4

P304

5299-5309

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P31

scholarly article

P356

10.1007/S12035-017-0763-4

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P433

http://www.w3.org/2001/XMLSchema#string

P478

http://www.w3.org/2001/XMLSchema#string

P50

Wuh-liang Hwu

Yin-Hsiu Chien

Ni-Chung Lee

P577

2017-09-11T00:00:00Z

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P6179

1091563517

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P698

28895054

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P921

gene therapy