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For: 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]
Number Citing Articles
1 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]
2 Pacitti D, Levene M, Garone C, Nirmalananthan N, Bax BE. Mitochondrial Neurogastrointestinal Encephalomyopathy: Into the Fourth Decade, What We Have Learned So Far. Front Genet 2018;9:669. [PMID: 30627136 DOI: 10.3389/fgene.2018.00669] [Cited by in Crossref: 17] [Cited by in F6Publishing: 17] [Article Influence: 4.3] [Reference Citation Analysis]
3 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]
4 Hirano M, Carelli V, De Giorgio R, Pironi L, Accarino A, Cenacchi G, D'Alessandro R, Filosto M, Martí R, Nonino F, Pinna AD, Baldin E, Bax BE, Bolletta A, Bolletta R, Boschetti E, Cescon M, D'Angelo R, Dotti MT, Giordano C, Gramegna LL, Levene M, Lodi R, Mandel H, Morelli MC, Musumeci O, Pugliese A, Scarpelli M, Siniscalchi A, Spinazzola A, Tal G, Torres-Torronteras J, Vignatelli L, Zaidman I, Zoller H, Rinaldi R, Zeviani M. Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): Position paper on diagnosis, prognosis, and treatment by the MNGIE International Network. J Inherit Metab Dis 2021;44:376-87. [PMID: 32898308 DOI: 10.1002/jimd.12300] [Cited by in Crossref: 9] [Cited by in F6Publishing: 8] [Article Influence: 4.5] [Reference Citation Analysis]
5 Levene M, Bain MD, Moran NF, Nirmalananthan N, Poulton J, Scarpelli M, Filosto M, Mandel H, MacKinnon AD, Fairbanks L, Pacitti D, Bax BE. Safety and Efficacy of Erythrocyte Encapsulated Thymidine Phosphorylase in Mitochondrial Neurogastrointestinal Encephalomyopathy. J Clin Med 2019;8:E457. [PMID: 30959750 DOI: 10.3390/jcm8040457] [Cited by in Crossref: 17] [Cited by in F6Publishing: 17] [Article Influence: 5.7] [Reference Citation Analysis]
6 Yadak R, Breur M, Bugiani M. Gastrointestinal Dysmotility in MNGIE: from thymidine phosphorylase enzyme deficiency to altered interstitial cells of Cajal. Orphanet J Rare Dis. 2019;14:33. [PMID: 30736844 DOI: 10.1186/s13023-019-1016-6] [Cited by in Crossref: 8] [Cited by in F6Publishing: 10] [Article Influence: 2.7] [Reference Citation Analysis]
7 López-gómez C, Samara Y, Hirano M, Martí R. 232nd ENMC International Workshop: Recommendations for treatment of mitochondrial DNA maintenance disorders. 16 – 18 June 2017, Heemskerk, The Netherlands. Neuromuscular Disorders 2022. [DOI: 10.1016/j.nmd.2022.05.008] [Reference Citation Analysis]
8 Yadak R, Boot MV, van Til NP, Cazals-Hatem D, Finkenstedt A, Bogaerts E, de Coo IF, Bugiani M. Transplantation, gene therapy and intestinal pathology in MNGIE patients and mice. BMC Gastroenterol 2018;18:149. [PMID: 30340467 DOI: 10.1186/s12876-018-0881-0] [Cited by in Crossref: 7] [Cited by in F6Publishing: 8] [Article Influence: 1.8] [Reference Citation Analysis]
9 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]
10 Meinders M, Shoemark D, Dobbe JGG, Streekstra GJ, Frayne J, Toye AM. Expression and Retention of Thymidine Phosphorylase in Cultured Reticulocytes as a Novel Treatment for MNGIE. Mol Ther Methods Clin Dev 2020;17:822-30. [PMID: 32368563 DOI: 10.1016/j.omtm.2020.03.029] [Cited by in Crossref: 1] [Cited by in F6Publishing: 1] [Article Influence: 0.5] [Reference Citation Analysis]
11 Cabrera-Pérez R, Vila-Julià F, Hirano M, Mingozzi F, Torres-Torronteras J, Martí R. Alpha-1-Antitrypsin Promoter Improves the Efficacy of an Adeno-Associated Virus Vector for the Treatment of Mitochondrial Neurogastrointestinal Encephalomyopathy. Hum Gene Ther 2019;30:985-98. [PMID: 30900470 DOI: 10.1089/hum.2018.217] [Cited by in Crossref: 8] [Cited by in F6Publishing: 6] [Article Influence: 2.7] [Reference Citation Analysis]
12 Hirano M, Emmanuele V, Quinzii CM. Emerging therapies for mitochondrial diseases. Essays Biochem 2018;62:467-81. [PMID: 29980632 DOI: 10.1042/EBC20170114] [Cited by in Crossref: 59] [Cited by in F6Publishing: 25] [Article Influence: 14.8] [Reference Citation Analysis]
13 Filosto M, Cotti Piccinelli S, Caria F, Gallo Cassarino S, Baldelli E, Galvagni A, Volonghi I, Scarpelli M, Padovani A. Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE-MTDPS1). J Clin Med 2018;7:E389. [PMID: 30373120 DOI: 10.3390/jcm7110389] [Cited by in Crossref: 14] [Cited by in F6Publishing: 10] [Article Influence: 3.5] [Reference Citation Analysis]