Title: | Long-Term Sustained Effect of Liver-Targeted Adeno-Associated Virus Gene Therapy for Mitochondrial Neurogastrointestinal Encephalomyopathy |
Journal : | Human gene therapy |
Authors: | Torres-Torronteras J, Author ; Cabrera-Pérez R ; Vila-Julia F ; Viscomi C ; Camara Y ; Hirano M ; Zeviani M ; Marti R |
Material Type: | Article |
Publication Date: | 26/02/2018 |
Languages: | English |
Keywords : | AAV2 vectors ; adenoassociated vectors ; gene therapy ; gène TYMP ; liver ; mitochondrial diseases ; MNGIE ; mouse model ; preclinical trials ; pyrimidic nucleosides and nucleotides ; therapeutic prospects ; thymidine ; thymidine phosphorylase |
Keywords: | AAV;MNGIE;liver/metabolic;mitochondria;thymidine phosphorylase; |
Pubmed / DOI : | DOI : 10.1089/hum.2017.133 / Pubmed : 29284302 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/29284302 |
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