widespread aav1- and aav2-mediated transgene expression in the nonhuman primate brain: implications for huntington's disease
Clicks: 236
ID: 187932
2016
Article Quality & Performance Metrics
Overall Quality
Not rated
Combines reader engagement with the AI quality analysis. This
article has not been analysed, so there is no overall score —
reader engagement is measured and shown alongside.
Reader Engagement
Steady Performance
30.0
/100
236 views
36 readers
AI Quality Assessment
Not analyzed
Readership in this journal
SteadyRanked #18 of 38 articles by views in international journal of zoology
Most read
Least read
Bar heights use a square-root scale.
Mint this article as an NFT
Not yet mintedCreate a permanent, verifiable on-chain record of this article on the Scimatic Network. The NFT is held in your Journament account, and you can withdraw it to your own wallet at any time.
5
SUSD
one-off · no wallet required
Abstract
Huntington's disease (HD) is caused by a toxic gain-of-function associated with the expression of the mutant huntingtin (htt) protein. Therefore, the use of RNA interference to inhibit Htt expression could represent a disease-modifying therapy. The potential of two recombinant adeno-associated viral vectors (AAV), AAV1 and AAV2, to transduce the cortico-striatal tissues that are predominantly affected in HD was explored. Green fluorescent protein was used as a reporter in each vector to show that both serotypes were broadly distributed in medium spiny neurons in the striatum and cortico-striatal neurons after infusion into the putamen and caudate nucleus of nonhuman primates (NHP), with AAV1-directed expression being slightly more robust than AAV2-driven expression. This study suggests that both serotypes are capable of targeting neurons that degenerate in HD, and it sets the stage for the advanced preclinical evaluation of an RNAi-based therapy for this disease.
| Reference Key |
hadaczek2016molecularwidespread
Use this key to autocite in the manuscript while using
SciMatic Manuscript Manager or Thesis Manager
|
|---|---|
| Authors | ;Piotr Hadaczek;Lisa Stanek;Agnieszka Ciesielska;Vivek Sudhakar;Lluis Samaranch;Philip Pivirotto;John Bringas;Catherine O'Riordan;Bryan Mastis;Waldy San Sebastian;John Forsayeth;Seng H Cheng;Krystof S Bankiewicz;Lamya S Shihabuddin |
| Journal | international journal of zoology |
| Year | 2016 |
| DOI |
10.1038/mtm.2016.37
|
| URL | |
| Keywords |
Citations
No citations found. To add a citation, contact the admin at info@scimatic.org
Comments
No comments yet. Be the first to comment on this article.