Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE-Reference-Cited by-同舟云学术

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Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE

Published:2018-03 Issue: Volume:8 Page:152-165
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Yadak Rana,Cabrera-Pérez Raquel,Torres-Torronteras Javier,Bugiani Marianna,Haeck Joost C.,Huston Marshall W.,Bogaerts Elly,Goffart Steffi,Jacobs Edwin H.,Stok Merel,Leonardelli Lorena,Biasco Luca,Verdijk Robert M.,Bernsen Monique R.,Ruijter George,Martí Ramon,Wagemaker Gerard,van Til Niek P.,de Coo Irenaeus F.M.

Funder

Join4energy

Ride4Kids

Sophia Foundation

Stichting NeMo

Spanish Instituto de Salud Carlos III

FEDER funds

Netherlands Health Research and Development Organization ZonMw

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference51 articles.

1. Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): a disease of two genomes;Hirano;Neurologist,2004

2. Late-onset MNGIE without peripheral neuropathy due to incomplete loss of thymidine phosphorylase activity;Massa;Neuromuscul. Disord.,2009

3. Thymidine and deoxyuridine accumulate in tissues of patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE);Valentino;FEBS Lett.,2007

4. Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): clinical, biochemical, and genetic features of an autosomal recessive mitochondrial disorder;Hirano;Neurology,1994

5. Limited dCTP availability accounts for mitochondrial DNA depletion in mitochondrial neurogastrointestinal encephalomyopathy (MNGIE);González-Vioque;PLoS Genet.,2011

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