Hematopoietic stem cell gene therapy leads to marked visceral organ improvements and a delayed onset of neurological abnormalities in the acid sphingomyelinase deficient mouse model of Niemann-Pick disease.

Hematopoietic stem cell gene therapy leads to marked visceral organ improvements and a delayed onset of neurological abnormalities in the acid sphingomyelinase deficient mouse model of Niemann-Pick disease.

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http://www.wikidata.org/entity/Q45872484

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Experimental neurotransplantation treatment for hereditary cerebellar ataxias Intracerebral transplantation of mesenchymal stem cells into acid sphingomyelinase-deficient mice delays the onset of neurological abnormalities and extends their life span.Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?Gene therapy progress and prospects: gene therapy of lysosomal storage disorders.Gene therapy for lipoprotein disorders.Gene Therapy for the Treatment of Neurological Disorders: Metabolic Disorders.SMPD1 Mutation Update: Database and Comprehensive Analysis of Published and Novel Variants.Types A and B Niemann-Pick disease.

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P2860

Hematopoietic stem cell gene therapy leads to marked visceral organ improvements and a delayed onset of neurological abnormalities in the acid sphingomyelinase deficient mouse model of Niemann-Pick disease.

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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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Hematopoietic stem cell gene t ...... model of Niemann-Pick disease.

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Hematopoietic stem cell gene t ...... model of Niemann-Pick disease.

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Hematopoietic stem cell gene t ...... model of Niemann-Pick disease.

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Hematopoietic stem cell gene t ...... model of Niemann-Pick disease.

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Hematopoietic stem cell gene t ...... model of Niemann-Pick disease.

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P2860

Molecular Cloning of the Acid Sphingomyelinase of the Mouse and the Organization and Complete Nucleotide Sequence of the Gene

Acid sphingomyelinase deficient mice: a model of types A and B Niemann-Pick disease

Structural organization and complete nucleotide sequence of the gene encoding human acid sphingomyelinase (SMPD1)

Zn2+-stimulated sphingomyelinase is secreted by many cell types and is a product of the acid sphingomyelinase gene

The metabolism of sphingomyelin. II. Evidence of an enzymatic deficiency in Niemann-Pick diseae.

A calcium responsive element that regulates expression of two calcium binding proteins in Purkinje cells.

Arylsulfatase B activities and glycosaminoglycan levels in retrovirally transduced mucopolysaccharidosis type VI cells. Prospects for gene therapy.

Banded krait minor-satellite (Bkm)-associated Y chromosome-specific repetitive DNA in mouse.

Replacement therapy for inherited enzyme deficiency: liver orthotopic transplantation in Niemann-Pick disease type A.

Retrovirus-mediated transfer of the human alpha-L-iduronidase cDNA into human hematopoietic progenitor cells leads to correction in trans of Hurler fibroblasts.

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gene therapy

Hematopoietic stem cell gene therapy leads to marked visceral organ improvements and a delayed onset of neurological abnormalities in the acid sphingomyelinase deficient mouse model of Niemann-Pick disease.

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P2860

P2860

Hematopoietic stem cell gene therapy leads to marked visceral organ improvements and a delayed onset of neurological abnormalities in the acid sphingomyelinase deficient mouse model of Niemann-Pick disease.

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