Triptolide T10 enhances AAV-mediated gene transfer in mice striatum.
Adeno-associated virus (AAV) mediated gene transfer has been demonstrated to be an effective approach for treating Parkinson's disease (PD). Triptolide T10 is a monomeric compound isolated from tripterygium wilfordii Hook.f. (Thunder God vine), a traditional Chinese herb for anti-inflammatory medications. In the present study, we co-administered T10 with recombinant AAV2 in SH-SY5Y human neuroblastoma cells and in the striatum of C57BL/6 mice, and then evaluated the AAV-mediated gene expression levels. The results have shown that T10 significantly augmented the expression of AAV-mediated gene in a dose-dependent fashion without detectable cytotoxicity. As growing evidence indicated that inflammation contributed to the progression of PD, and the anti-inflammatory effect of T10 was shown in our previous studies, our data of T10 to enhance AAV transduction suggest that T10 might be potentially used as a facilitating reagent for the AAV gene therapy applications in neurodegenerative diseases.
Ren
Xinmiao
X
Department of Physiology and Key Laboratory of the Neurodegenerative Disorders of the Chinese Ministry of Education, Capital Medical University, Youanmen, Beijing 100069, PR China.
Zhang
Ting
T
Hu
Jing
J
Ding
Wei
W
Wang
Xiaomin
X
D000818:Animals; D000893:Anti-Inflammatory Agents / Q000494:pharmacology*; D002460:Cell Line; D002470:Cell Survival / Q000187:drug effects; D003342:Corpus Striatum / Q000187:drug effects* / Q000378:metabolism; D000229:Dependovirus / Q000235:genetics*; D004224:Diterpenes / Q000494:pharmacology*; D004365:Drugs, Chinese Herbal / Q000494:pharmacology; D004852:Epoxy Compounds / Q000494:pharmacology; D018014:Gene Transfer Techniques; D049452:Green Fluorescent Proteins / Q000096:biosynthesis / Q000235:genetics; D006801:Humans; D008156:Luciferases / Q000235:genetics / Q000378:metabolism; D008297:Male; D051379:Mice; D008810:Mice, Inbred C57BL; D018696:Neuroprotective Agents / Q000494:pharmacology*; D010616:Phenanthrenes / Q000494:pharmacology*; D014161:Transduction, Genetic; D019076:Transgenes
D000893:Anti-Inflammatory Agents; D004224:Diterpenes; D004365:Drugs, Chinese Herbal; D004852:Epoxy Compounds; D018696:Neuroprotective Agents; D010616:Phenanthrenes; C485184:enhanced green fluorescent protein; D049452:Green Fluorescent Proteins; C001899:triptolide; D008156:Luciferases