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For: Torres-Torronteras J, Viscomi C, Cabrera-Pérez R, Cámara Y, Di Meo I, Barquinero J, Auricchio A, Pizzorno G, Hirano M, Zeviani M, Martí R. Gene therapy using a liver-targeted AAV vector restores nucleoside and nucleotide homeostasis in a murine model of MNGIE. Mol Ther 2014;22:901-7. [PMID: 24448160 DOI: 10.1038/mt.2014.6] [Cited by in Crossref: 41] [Cited by in F6Publishing: 34] [Article Influence: 5.1] [Reference Citation Analysis]
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13 Viscomi C, Bottani E, Zeviani M. Emerging concepts in the therapy of mitochondrial disease. Biochim Biophys Acta 2015;1847:544-57. [PMID: 25766847 DOI: 10.1016/j.bbabio.2015.03.001] [Cited by in Crossref: 75] [Cited by in F6Publishing: 65] [Article Influence: 10.7] [Reference Citation Analysis]
14 Torres-Torronteras J, Cabrera-Pérez R, Vila-Julià F, Viscomi C, Cámara Y, Hirano M, Zeviani M, Martí R. Long-Term Sustained Effect of Liver-Targeted Adeno-Associated Virus Gene Therapy for Mitochondrial Neurogastrointestinal Encephalomyopathy. Hum Gene Ther 2018;29:708-18. [PMID: 29284302 DOI: 10.1089/hum.2017.133] [Cited by in Crossref: 20] [Cited by in F6Publishing: 18] [Article Influence: 5.0] [Reference Citation Analysis]
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18 Parés M, Fornaguera C, Vila-Julià F, Oh S, Fan SHY, Tam YK, Comes N, Vidal F, Martí R, Borrós S, Barquinero J. Preclinical Assessment of a Gene-Editing Approach in a Mouse Model of Mitochondrial Neurogastrointestinal Encephalomyopathy. Hum Gene Ther 2021;32:1210-23. [PMID: 34498979 DOI: 10.1089/hum.2021.152] [Reference Citation Analysis]
19 Yadak R, Cabrera-Pérez R, Torres-Torronteras J, Bugiani M, Haeck JC, Huston MW, Bogaerts E, Goffart S, Jacobs EH, Stok M, Leonardelli L, Biasco L, Verdijk RM, Bernsen MR, Ruijter G, Martí R, Wagemaker G, van Til NP, de Coo IFM. Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE. Mol Ther Methods Clin Dev 2018;8:152-65. [PMID: 29687034 DOI: 10.1016/j.omtm.2018.01.001] [Cited by in Crossref: 13] [Cited by in F6Publishing: 12] [Article Influence: 3.3] [Reference Citation Analysis]
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21 Vila-Julià F, Cabrera-Pérez R, Cámara Y, Molina-Berenguer M, Lope-Piedrafita S, Hirano M, Mingozzi F, Torres-Torronteras J, Martí R. Efficacy of adeno-associated virus gene therapy in a MNGIE murine model enhanced by chronic exposure to nucleosides. EBioMedicine 2020;62:103133. [PMID: 33232869 DOI: 10.1016/j.ebiom.2020.103133] [Cited by in Crossref: 2] [Cited by in F6Publishing: 2] [Article Influence: 1.0] [Reference Citation Analysis]
22 Nightingale H, Pfeffer G, Bargiela D, Horvath R, Chinnery PF. Emerging therapies for mitochondrial disorders. Brain 2016;139:1633-48. [PMID: 27190030 DOI: 10.1093/brain/aww081] [Cited by in Crossref: 38] [Cited by in F6Publishing: 32] [Article Influence: 6.3] [Reference Citation Analysis]
23 Torres-Torronteras J, Cabrera-Pérez R, Barba I, Costa C, de Luna N, Andreu AL, Barquinero J, Hirano M, Cámara Y, Martí R. Long-Term Restoration of Thymidine Phosphorylase Function and Nucleoside Homeostasis Using Hematopoietic Gene Therapy in a Murine Model of Mitochondrial Neurogastrointestinal Encephalomyopathy. Hum Gene Ther 2016;27:656-67. [PMID: 27004974 DOI: 10.1089/hum.2015.160] [Cited by in Crossref: 20] [Cited by in F6Publishing: 17] [Article Influence: 3.3] [Reference Citation Analysis]
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25 Yadak R, Sillevis Smitt P, van Gisbergen MW, van Til NP, de Coo IF. Mitochondrial Neurogastrointestinal Encephalomyopathy Caused by Thymidine Phosphorylase Enzyme Deficiency: From Pathogenesis to Emerging Therapeutic Options. Front Cell Neurosci 2017;11:31. [PMID: 28261062 DOI: 10.3389/fncel.2017.00031] [Cited by in Crossref: 28] [Cited by in F6Publishing: 21] [Article Influence: 5.6] [Reference Citation Analysis]
26 Ramón J, Vila-Julià F, Molina-Granada D, Molina-Berenguer M, Melià MJ, García-Arumí E, Torres-Torronteras J, Cámara Y, Martí R. Therapy Prospects for Mitochondrial DNA Maintenance Disorders. Int J Mol Sci 2021;22:6447. [PMID: 34208592 DOI: 10.3390/ijms22126447] [Reference Citation Analysis]
27 Bottani E, Lamperti C, Prigione A, Tiranti V, Persico N, Brunetti D. Therapeutic Approaches to Treat Mitochondrial Diseases: "One-Size-Fits-All" and "Precision Medicine" Strategies. Pharmaceutics 2020;12:E1083. [PMID: 33187380 DOI: 10.3390/pharmaceutics12111083] [Cited by in Crossref: 10] [Cited by in F6Publishing: 10] [Article Influence: 5.0] [Reference Citation Analysis]
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30 Dionisi-Vici C, Diodato D, Torre G, Picca S, Pariante R, Giuseppe Picardo S, Di Meo I, Rizzo C, Tiranti V, Zeviani M, De Ville De Goyet J. Liver transplant in ethylmalonic encephalopathy: a new treatment for an otherwise fatal disease. Brain. 2016;139:1045-1051. [PMID: 26917598 DOI: 10.1093/brain/aww013] [Cited by in Crossref: 49] [Cited by in F6Publishing: 38] [Article Influence: 8.2] [Reference Citation Analysis]
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