Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.

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Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.

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2016 nî lūn-bûn

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2016年の論文

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2016年論文

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2016年論文

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2016年論文

@zh-hk

2016年論文

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2016年論文

@zh-tw

2016年论文

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2016年论文

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2016年论文

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Combination Therapies for Lyso ...... ex Answer to a Simple Problem.

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Combination Therapies for Lyso ...... ex Answer to a Simple Problem.

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Combination Therapies for Lyso ...... ex Answer to a Simple Problem.

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P1476

Combination Therapies for Lyso ...... ex Answer to a Simple Problem.

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P2093

Shannon L Macauley

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The pharmacological chaperone AT2220 increases the specific activity and lysosomal delivery of mutant acid alpha-glucosidase, and promotes glycogen reduction in a transgenic mouse model of Pompe disease

A mouse model of classical late-infantile neuronal ceroid lipofuscinosis based on targeted disruption of the CLN2 gene results in a loss of tripeptidyl-peptidase I activity and progressive neurodegeneration

Substrate reduction augments the efficacy of enzyme therapy in a mouse model of Fabry disease

Functions of lysosomes

Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy.

Bone marrow transplantation as effective treatment of central nervous system disease in globoid cell leukodystrophy, metachromatic leukodystrophy, adrenoleukodystrophy, mannosidosis, fucosidosis, aspartylglucosaminuria, Hurler, Maroteaux-Lamy, and S

The defect in Hurler and Hunter syndromes. II. Deficiency of specific factors involved in mucopolysaccharide degradation

Therapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIB

Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis.

Common and uncommon pathogenic cascades in lysosomal storage diseases

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13 Suppl 1

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Shannon L Macauley

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2016-06-01T00:00:00Z

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Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.

about

Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.

description

name

type

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prefLabel

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P1476

P2093

P2860

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P478

P50

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P698

P932

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P2093

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