Recombinant Adeno Associated Viral (AAV) vector type 9 delivery of Ex1-Q138-mutant huntingtin in the rat striatum as a short-time model for in vivo studies in drug discovery
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- 作者：Ilaria Ceccarelli1 ; Pasquale Fiengo2 ; Rosaria Remelli ; Vincenzo Miragliotta3 ; Lara Rossini4 ; Irene Biotti ; Alessandra Cappelli ; Lara Petricca5 ; Salvatore La Rosa6 ; Andrea Caricasole5 ; Giuseppe Pollio ; Carla Scali ; carla.scali@outlook.com" class="auth_mail" title="E-mail the corresponding author
- 关键词：HD ; Huntington's disease ; mHTT ; mutant Huntingtin ; rAAV ; recombinant Adeno Associated Virus ; LV ; lentivirus ; tNAA ; total N-Acetyl Aspartate ; Myo-Ins ; myoinositol ; GFP ; green fluorescent protein ; NeuN ; neuronal nuclei ; DARPP-32 ; dopamine- and cyclic AMP-regulated phosphoprotein ; ChAT ; choline acetyl transferase ; GFAP ; glial fibrillary acidic protein ; Iba-1 ; ionized calcium binding adaptor molecule 1 ; PBST ; phosphate buffered saline-Triton-X100 ; BSA ; bovine serum albumin ; DAB ; diaminobenzidine ; RO
- 刊名：Neurobiology of Disease
- 出版年：2016
- 出版时间：February 2016
- 年：2016
- 卷：86
- 期：Complete
- 页码：41-51
- 全文大小：1702 K
文摘
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rAAV2/9-Exon 1 HTT 17 or 138 CAG was used to create a new rat model of HD.
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Exon 1 HTT-Q138 induced neurodegeneration and neuroinflammation in the striatum.
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Genomic analysis and H1-MRS study revealed high association with human HD hallmarks.
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We pharmacologically validated our model using cystamine.
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Our model provide useful tool for drug discovery process.