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Title
Lentivirus-mediated gene therapy for Fabry disease
Authors
Keywords
-
Journal
Nature Communications
Volume 12, Issue 1, Pages -
Publisher
Springer Science and Business Media LLC
Online
2021-02-25
DOI
10.1038/s41467-021-21371-5
References
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Note: Only part of the references are listed.- Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study
- (2019) Francesca Ferrua et al. Lancet Haematology
- Comparison of Cardiac Magnetic Resonance Imaging and Echocardiography in Assessment of Left Ventricular Hypertrophy in Fabry Disease
- (2018) Hassan Hazari et al. CANADIAN JOURNAL OF CARDIOLOGY
- Effectiveness of plasma lyso-Gb3 as a biomarker for selecting high-risk patients with Fabry disease from multispecialty clinics for genetic analysis
- (2018) Hiroki Maruyama et al. GENETICS IN MEDICINE
- Fabry disease revisited: Management and treatment recommendations for adult patients
- (2018) Alberto Ortiz et al. MOLECULAR GENETICS AND METABOLISM
- European expert consensus statement on therapeutic goals in Fabry disease
- (2018) Christoph Wanner et al. MOLECULAR GENETICS AND METABOLISM
- Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
- (2018) Alexis A. Thompson et al. NEW ENGLAND JOURNAL OF MEDICINE
- Lysosomal storage diseases
- (2018) Frances M. Platt et al. Nature Reviews Disease Primers
- Gene Therapy in a Patient with Sickle Cell Disease
- (2017) Jean-Antoine Ribeil et al. NEW ENGLAND JOURNAL OF MEDICINE
- Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34 + Cells for Correction of Fabry Disease
- (2017) Ju Huang et al. Molecular Therapy-Methods & Clinical Development
- Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat
- (2016) Dominique P. Germain et al. NEW ENGLAND JOURNAL OF MEDICINE
- Urinary biomarker investigation in children with Fabry disease using tandem mass spectrometry
- (2015) Christiane Auray-Blais et al. CLINICA CHIMICA ACTA
- Fabry disease and enzyme replacement therapy in classic patients with same mutation: different formulations - different outcome?
- (2015) J. Politei et al. CLINICAL GENETICS
- Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document
- (2015) Marieke Biegstraaten et al. Orphanet Journal of Rare Diseases
- Multiplex Tandem Mass Spectrometry Analysis of Novel Plasma Lyso-Gb3-Related Analogues in Fabry Disease
- (2014) Michel Boutin et al. ANALYTICAL CHEMISTRY
- Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain
- (2013) Saskia M Rombach et al. Orphanet Journal of Rare Diseases
- Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
- (2013) A. Biffi et al. SCIENCE
- Urinary Globotriaosylsphingosine-Related Biomarkers for Fabry Disease Targeted by Metabolomics
- (2012) Christiane Auray-Blais et al. ANALYTICAL CHEMISTRY
- Multiplex Analysis of Novel Urinary Lyso-Gb3-Related Biomarkers for Fabry Disease by Tandem Mass Spectrometry
- (2012) Pamela Lavoie et al. ANALYTICAL CHEMISTRY
- Promoter-specific lentivectors for long-term, cardiac-directed therapy of Fabry disease
- (2010) Chyan-Jang Lee et al. Journal of Cardiology
- Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome
- (2010) Kaan Boztug et al. NEW ENGLAND JOURNAL OF MEDICINE
- Life expectancy and cause of death in males and females with Fabry disease: Findings from the Fabry Registry
- (2009) Stephen Waldek et al. GENETICS IN MEDICINE
- Elevated globotriaosylsphingosine is a hallmark of Fabry disease
- (2008) J. M. Aerts et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
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