Title: | Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9 |
Journal : | Cell, 170, 5 |
Authors: | Batra R, Author ; Nelles DA ; Pirie E ; Blue SM ; Marina RJ ; Wang H ; Chaim IA ; Thomas JD ; Zhang N ; Nguyen V ; Aigner S ; Markmiller S ; Xia G ; Corbett KD ; Swanson MS ; Yeo GW |
Material Type: | Article |
Publication Date: | 2017 |
Size: | p 899 |
Languages: | English |
Keywords : | cell cultures ; microsatellites ; myoblast ; myotonic dystrophy type 1 ; repeated sequence amplification ; RNA-targeting Cas9 ; splicing ; therapeutic prospects ; toxic RNA |
Keywords: | ALS;CRISPR;Huntington’s disease;RNA-targeting Cas9;adeno-associated virus;gene therapy;microsatellite repeat expansion;minimal Cas9;myotonic dystrophy; |
Abstract: |
Comment in:
A Therapeutic Double Whammy: Transcriptional or Post-transcriptional Suppression of Microsatellite Repeat Toxicity by Cas9. [Mol Cell. 2017] |
Pubmed / DOI : | DOI : 10.1016/j.cell.2017.07.010 / Pubmed : 28803727 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/28803727 |