Gene Therapy Corrects Mitochondrial Dysfunction in Hematopoietic Progenitor Cells and Fibroblasts from Coq9R239X Mice
Publication Date
June 24, 2016
Journal
PLOS ONE
Authors
Eliana Barriocanal Casado, Cristina Cueto Ureña, Karim Benabdellah, Alejandra Gutiérrez Guerrero, et al
Volume
11
Issue
6
Pages
e0158344
DOI
https://dx.plos.org/10.1371/journal.pone.0158344
Publisher URL
http://journals.plos.org/plosone/article?id=10.1371%2Fjournal.pone.0158344
Scopus
84976499618
Mendeley
http://www.mendeley.com/research/gene-therapy-corrects-mitochondrial-dysfunction-hematopoietic-progenitor-cells-fibroblasts-coq9r239x
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Mendeley | Further Information

{"title"=>"Gene therapy corrects mitochondrial dysfunction in hematopoietic progenitor cells and fibroblasts from Coq9R239X mice", "type"=>"journal", "authors"=>[{"first_name"=>"Eliana", "last_name"=>"Barriocanal-Casado", "scopus_author_id"=>"57190018288"}, {"first_name"=>"Cristina", "last_name"=>"Cueto-Ureña", "scopus_author_id"=>"56278018700"}, {"first_name"=>"Karim", "last_name"=>"Benabdellah", "scopus_author_id"=>"6506137846"}, {"first_name"=>"Alejandra", "last_name"=>"Gutiérrez-Guerrero", "scopus_author_id"=>"56085230600"}, {"first_name"=>"Marién", "last_name"=>"Cobo", "scopus_author_id"=>"54379542500"}, {"first_name"=>"Agustín", "last_name"=>"Hidalgo-Gutiérrez", "scopus_author_id"=>"57190016412"}, {"first_name"=>"Juan Jose", "last_name"=>"Rodríguez-Sevilla", "scopus_author_id"=>"57190013180"}, {"first_name"=>"Francisco", "last_name"=>"Martín", "scopus_author_id"=>"57192048020"}, {"first_name"=>"Luis C.", "last_name"=>"López", "scopus_author_id"=>"25954462600"}], "year"=>2016, "source"=>"PLoS ONE", "identifiers"=>{"pui"=>"610977649", "issn"=>"19326203", "doi"=>"10.1371/journal.pone.0158344", "scopus"=>"2-s2.0-84976499618", "pmid"=>"27341668", "sgr"=>"84976499618", "isbn"=>"1932-6203 (Electronic)\\r1932-6203 (Linking)"}, "id"=>"4aa32e4a-816b-3bc7-9984-a493be9bbc4b", "abstract"=>"© 2016 Barriocanal-Casado et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.Recent clinical trials have shown that in vivo and ex vivo gene therapy strategies can be an option for the treatment of several neurological disorders. Both strategies require efficient and safe vectors to 1) deliver the therapeutic gene directly into the CNS or 2) to genetically modify stem cells that will be used as Trojan horses for the systemic delivery of the therapeutic protein. A group of target diseases for these therapeutic strategies are mitochondrial encephalopathies due to mutations in nuclear DNA genes. In this study, we have developed a lentiviral vector (CCoq9WP) able to overexpress Coq9 mRNA and COQ9 protein in mouse embryonic fibroblasts (MEFs) and hematopoietic progenitor cells (HPCs) from Coq9R239X mice, an animal model of mitochondrial encephalopathy due to primary Coenzyme Q (CoQ) deficiency. Ectopic over-expression of Coq9 in both cell types restored the CoQ biosynthetic pathway and mitochondrial function, improving the fitness of the transduced cells. These results show the potential of the CCoq9WP lentiviral vector as a tool for gene therapy to treat mitochondrial encephalopathies.", "link"=>"http://www.mendeley.com/research/gene-therapy-corrects-mitochondrial-dysfunction-hematopoietic-progenitor-cells-fibroblasts-coq9r239x", "reader_count"=>7, "reader_count_by_academic_status"=>{"Professor > Associate Professor"=>1, "Student > Master"=>2, "Student > Bachelor"=>2, "Lecturer"=>1, "Professor"=>1}, "reader_count_by_user_role"=>{"Professor > Associate Professor"=>1, "Student > Master"=>2, "Student > Bachelor"=>2, "Lecturer"=>1, "Professor"=>1}, "reader_count_by_subject_area"=>{"Biochemistry, Genetics and Molecular Biology"=>4, "Agricultural and Biological Sciences"=>2, "Medicine and Dentistry"=>1}, "reader_count_by_subdiscipline"=>{"Medicine and Dentistry"=>{"Medicine and Dentistry"=>1}, "Agricultural and Biological Sciences"=>{"Agricultural and Biological Sciences"=>2}, "Biochemistry, Genetics and Molecular Biology"=>{"Biochemistry, Genetics and Molecular Biology"=>4}}, "group_count"=>1}

Scopus | Further Information

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