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Compound heterozygous FXN mutations and clinical outcome in friedreich ataxia

Annals of neurology, 2016-03, Vol.79 (3), p.485-495

2016

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FXN methylation predicts expression and clinical outcome in Friedreich ataxia

Annals of neurology, 2012-04, Vol.71 (4), p.487-497

2012

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Epigenetic modifications in trinucleotide repeat diseases

Trends in molecular medicine, 2013-11, Vol.19 (11), p.655-663

2013

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Zinc-regulated DNA binding of the yeast Zap1 zinc-responsive activator

PloS one, 2011-07, Vol.6 (7), p.e22535-e22535

2011

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Generation of induced pluripotent stem cell lines from Friedreich ataxia patients

Stem cell reviews, 2011-09, Vol.7 (3), p.703-713

2011

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An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation

Blood, 2008-02, Vol.111 (4), p.1866-1875

2008

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HFE p.C282Y heterozygosity is associated with earlier disease onset in Friedreich ataxia

Movement disorders, 2014-06, Vol.29 (7), p.940-943

2014

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An open-label trial in Friedreich ataxia suggests clinical benefit with high-dose resveratrol, without effect on frataxin levels

Journal of neurology, 2015-05, Vol.262 (5), p.1344-1353

2015

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Cell and gene therapy for Friedreich ataxia: progress to date

Human gene therapy, 2014-08, Vol.25 (8), p.684-693

2014

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Friedreich's ataxia induced pluripotent stem cell-derived cardiomyocytes display electrophysiological abnormalities and calcium handling deficiency

Aging (Albany, NY.), 2017-05, Vol.9 (5), p.1440-1452

2017

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Suppression of Clonal Dominance in Cultured Human Lymphoid Cells by Addition of the cHS4 Insulator to a Lentiviral Vector

Molecular therapy, 2007-04, Vol.15 (4), p.801-809

2007

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Beyond loss of frataxin: the complex molecular pathology of Friedreich ataxia

Discovery medicine, 2014-01, Vol.17 (91), p.25

2014

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Toxicity of linoleic acid hydroperoxide to Saccharomyces cerevisiae: involvement of a respiration-related process for maximal sensitivity and adaptive response

Journal of bacteriology, 1998-02, Vol.180 (3), p.483-490

1998

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Characterising the neuropathology and neurobehavioural phenotype in Friedreich ataxia: a systematic review

Advances in experimental medicine and biology, 2012-01, Vol.769, p.169

2012

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Multisite formative assessment for the Pathways study to prevent obesity in American Indian schoolchildren

The American journal of clinical nutrition, 1999-04, Vol.69 (4 Suppl), p.767S-772S

1999

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A novel deletion–insertion mutation identified in exon 3 of FXN in two siblings with a severe Friedreich ataxia phenotype

Neurogenetics, 2011-11, Vol.12 (4), p.307-313

2011

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A chromatin insulator blocks interactions between globin regulatory elements and cellular promoters in erythroid cells

Blood cells, molecules, & diseases, 2007-11, Vol.39 (3), p.221-228

2007

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A clinical trial of the effects of dietary patterns on blood pressure

The New England journal of medicine, 1997-04, Vol.336 (16), p.1117-1124

1997

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A dual role for zinc fingers in both DNA binding and zinc sensing by the Zap1 transcriptional activator

The EMBO journal, 2000-07, Vol.19 (14), p.3704-3713

2000

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1058. Suppression of Clonal Dominance in Cultured Human Lymphoid Cells by Addition of the 5′cHS4 Insulator to a Lentiviral Vector

Molecular therapy, 2006-05, Vol.13 (S1), p.S405-S406

2006

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Mapping the DNA Binding Domain of the Zap1 Zinc-responsive Transcriptional Activator

The Journal of biological chemistry, 2000-05, Vol.275 (21), p.16160-16166

2000

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The epidemic of obesity in American Indian communities and the need for childhood obesity-prevention programs

The American journal of clinical nutrition, 1999-04, Vol.69 (4 Suppl), p.747S-754S

1999

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68. Evidence for Correction of Mutations in the c-Kit Gene by rAAV Mediated Gene Targeting in W/Wv Mice

Molecular therapy, 2006-05, Vol.13 (S1), p.S29

2006

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Body composition and overweight prevalence in 1704 schoolchildren from 7 American Indian communities

The American journal of clinical nutrition, 2003-08, Vol.78 (2), p.308-312

2003

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374. Developing Bone Marrow Transplant and Lentiviral Vectors to Treat Friedreich Ataxia

Molecular therapy, 2016-05, Vol.24, p.S149-S150

2016

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Compound heterozygous FXN mutations and clinical outcome in friedreich ataxia

FXN methylation predicts expression and clinical outcome in Friedreich ataxia

Epigenetic modifications in trinucleotide repeat diseases

Zinc-regulated DNA binding of the yeast Zap1 zinc-responsive activator

Generation of induced pluripotent stem cell lines from Friedreich ataxia patients

An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation

HFE p.C282Y heterozygosity is associated with earlier disease onset in Friedreich ataxia

An open-label trial in Friedreich ataxia suggests clinical benefit with high-dose resveratrol, without effect on frataxin levels

Cell and gene therapy for Friedreich ataxia: progress to date

Friedreich's ataxia induced pluripotent stem cell-derived cardiomyocytes display electrophysiological abnormalities and calcium handling deficiency

Suppression of Clonal Dominance in Cultured Human Lymphoid Cells by Addition of the cHS4 Insulator to a Lentiviral Vector

Beyond loss of frataxin: the complex molecular pathology of Friedreich ataxia

Toxicity of linoleic acid hydroperoxide to Saccharomyces cerevisiae: involvement of a respiration-related process for maximal sensitivity and adaptive response

Characterising the neuropathology and neurobehavioural phenotype in Friedreich ataxia: a systematic review

Multisite formative assessment for the Pathways study to prevent obesity in American Indian schoolchildren

A novel deletion–insertion mutation identified in exon 3 of FXN in two siblings with a severe Friedreich ataxia phenotype

A chromatin insulator blocks interactions between globin regulatory elements and cellular promoters in erythroid cells

A clinical trial of the effects of dietary patterns on blood pressure

A dual role for zinc fingers in both DNA binding and zinc sensing by the Zap1 transcriptional activator

1058. Suppression of Clonal Dominance in Cultured Human Lymphoid Cells by Addition of the 5′cHS4 Insulator to a Lentiviral Vector

Mapping the DNA Binding Domain of the Zap1 Zinc-responsive Transcriptional Activator

The epidemic of obesity in American Indian communities and the need for childhood obesity-prevention programs

68. Evidence for Correction of Mutations in the c-Kit Gene by rAAV Mediated Gene Targeting in W/Wv Mice

Body composition and overweight prevalence in 1704 schoolchildren from 7 American Indian communities

374. Developing Bone Marrow Transplant and Lentiviral Vectors to Treat Friedreich Ataxia

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