(Q34483749)

English

Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice

scholarly article

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scholarly article

1 reference

Europe PubMed Central

2 August 2017

Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice (English)

1 reference

Europe PubMed Central

2 August 2017

1

1 reference

Europe PubMed Central

2 August 2017

author name string

Han IP

2

1 reference

Europe PubMed Central

2 August 2017

Limaye A

3

1 reference

Europe PubMed Central

2 August 2017

Xu R

4

1 reference

Europe PubMed Central

2 August 2017

Gelderman MP

5

1 reference

Europe PubMed Central

2 August 2017

Zerfas P

6

1 reference

Europe PubMed Central

2 August 2017

Tirumalai K

7

1 reference

Europe PubMed Central

2 August 2017

Murray GJ

8

1 reference

Europe PubMed Central

2 August 2017

During MJ

9

1 reference

Europe PubMed Central

2 August 2017

Brady RO

10

1 reference

Europe PubMed Central

2 August 2017

Qasba P

11

1 reference

Europe PubMed Central

2 August 2017

language of work or name

0 references

publication date

1 February 2001

1 reference

Europe PubMed Central

2 August 2017

number of pages

6

1 reference

based on heuristic

inferred from page(s)

Proceedings of the National Academy of Sciences of the United States of America

1 reference

Europe PubMed Central

2 August 2017

98

1 reference

Europe PubMed Central

2 August 2017

2676-2681

1 reference

Europe PubMed Central

2 August 2017

5

1 reference

Europe PubMed Central

2 August 2017

Remission in models of type 1 diabetes by gene therapy using a single-chain insulin analogue

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Reduction of globotriaosylceramide in Fabry disease mice by substrate deprivation

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Long-term enzyme correction and lipid reduction in multiple organs of primary and secondary transplanted Fabry mice receiving transduced bone marrow cells

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

A recombinant baculovirus-expressed Plasmodium falciparum receptor-binding domain of erythrocyte binding protein EBA-175 biologically mimics native protein.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Infusion of alpha-galactosidase A reduces tissue globotriaosylceramide storage in patients with Fabry disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Correction of enzymatic and lysosomal storage defects in Fabry mice by adenovirus-mediated gene transfer

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Aging accentuates and bone marrow transplantation ameliorates metabolic defects in Fabry disease mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

alpha-Galactosidase A deficient mice: a model of Fabry disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Adenovirus-mediated gene transfer and expression of human beta-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Immunofluorescence imaging diagnosis of Fabry heterozygotes using confocal laser scanning microscopy.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Retroviral coexpression of a multidrug resistance gene (MDR1) and human alpha-galactosidase A for gene therapy of Fabry disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Human alpha-galactosidase A: nucleotide sequence of a cDNA clone encoding the mature enzyme

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Lysosomal enzymes and their receptors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Structural organization of the human alpha-galactosidase A gene: further evidence for the absence of a 3' untranslated region

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Mannose 6-phosphate receptors and lysosomal enzyme targeting

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Biochemical and clinical analysis of accumulated glycolipids in symptomatic heterozygotes of angiokeratoma corporis diffusum (Fabry's disease) in comparison with hemizygotes

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Lysosomal storage diseases

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Characteristics of a human cell line transformed by DNA from human adenovirus type 5

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Quantitative analysis of plasma neutral glycosphingolipids by high performance liquid chromatography of their perbenzoyl derivatives

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Electrophoretic transfer of proteins from polyacrylamide gels to nitrocellulose sheets: procedure and some applications

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Purification and properties of the two major isozymes of alpha-galactosidase from human placenta

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

6 July 2018

Structural organization and expression of the mouse gene encoding alpha-galactosidase A.

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.051634498

21 January 2018

Circulating alpha-galactosidase A derived from transduced bone marrow cells: relevance for corrective gene transfer for Fabry disease.

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.051634498

21 January 2018

Route of administration determines induction of T-cell-independent humoral responses to adeno-associated virus vectors.

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.051634498

21 January 2018

Efficiency of adenovirus-mediated gene transfer into hepatocytes by liver asanguineous perfusion method

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

27 October 2018

Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30197

27 October 2018

Enzymatic Defect in Fabry's Disease

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.051634498

21 January 2018

Replacement Therapy for Inherited Enzyme Deficiency

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.051634498

21 January 2018

An oral vaccine against NMDAR1 with efficacy in experimental stroke and epilepsy

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.051634498

21 January 2018

Fabry's disease: absence of storage as a feature of liver sinus endothelium

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.051634498

21 January 2018

Concentrations of glycosyl ceramides in plasma and red cells in Fabry's disease, a glycolipid lipidosis

1 reference

https://pubmed.ncbi.nlm.nih.gov/11226298

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

IMPROVED PROCEDURE FOR THE EXTRACTION OF LIPIDS FROM HUMAN ERYTHROCYTES

1 reference

https://pubmed.ncbi.nlm.nih.gov/11226298

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1073/PNAS.051634498

1 reference

Europe PubMed Central

2 August 2017

2001PNAS...98.2676J

0 references

1 reference

Europe PubMed Central

2 August 2017

1 reference

Europe PubMed Central

2 August 2017

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