(Q41229793)

28 September 2017

Disease modeling and lentiviral gene transfer in patient-specific induced pluripotent stem cells from late-onset Pompe disease patient (English)

1 reference

28 September 2017

1 reference

Yohei Sato

1

1 reference

28 September 2017

Hiroshi Kobayashi

2

1 reference

28 September 2017

Takashi Higuchi

3

1 reference

28 September 2017

Yohta Shimada

4

1 reference

28 September 2017

Takumi Era

5

1 reference

28 September 2017

Shigemi Kimura

6

1 reference

28 September 2017

Yoshikatsu Eto

7

1 reference

28 September 2017

Hiroyuki Ida

8

1 reference

28 September 2017

Toya Ohashi

9

1 reference

28 September 2017

8 July 2015

1 reference

Molecular Therapy. Methods & Clinical Development

28 September 2017

1 reference

28 September 2017

2

1 reference

28 September 2017

15023

1 reference

Hypertrophic cardiomyopathy in pompe disease is not limited to the classic infantile-onset phenotype

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

Stem cell therapy. Use of differentiated pluripotent stem cells as replacement therapy for treating disease

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

New frontier in regenerative medicine: site-specific gene correction in patient-specific induced pluripotent stem cells

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

Induced pluripotent stem cell-derived cardiomyocytes in studies of inherited arrhythmias

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

Pathogenic mutation of ALK2 inhibits induced pluripotent stem cell reprogramming and maintenance: mechanisms of reprogramming and strategy for drug identification

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

Generation of induced pluripotent stem cells from human nasal epithelial cells using a Sendai virus vector

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

Autopsy findings in late-onset Pompe disease: a case report and systematic review of the literature

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

Cardiovascular abnormalities in late-onset Pompe disease and response to enzyme replacement therapy

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

Suppression of autophagy permits successful enzyme replacement therapy in a lysosomal storage disorder--murine Pompe disease

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

A randomized study of alglucosidase alfa in late-onset Pompe's disease

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

Induction of pluripotent stem cells from adult human fibroblasts by defined factors

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

Pompe disease in infants and children

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

Transplantation of genetically corrected human iPSC-derived progenitors in mice with limb-girdle muscular dystrophy.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

Human Pompe disease-induced pluripotent stem cells for pathogenesis modeling, drug testing and disease marker identification

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

Neonatal gene transfer using lentiviral vector for murine Pompe disease: long-term expression and glycogen reduction

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

alpha-Glucosidase deficiency in generalized glycogenstorage disease (Pompe's disease).

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4495721

A small molecule that promotes cardiac differentiation of human pluripotent stem cells under defined, cytokine- and xeno-free conditions

17 June 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/26199952

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1038/MTM.2015.23

1 reference

release_2qnuh5wemnaqfccjzasjxnbewy

28 September 2017

1 reference

Fatcat

https://api.fatcat.wiki/v0/release/2qnuh5wemnaqfccjzasjxnbewy

mapped directly with Wikidata item

24 November 2022

based on heuristic

1 reference

28 September 2017

PubMed publication ID

26199952

1 reference