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Modulating FKBP5/FKBP51 and autophagy lowers HTT (huntingtin) levels
Autophagy. 2021 May 24:1-22. doi: 10.1080/15548627.2021.1904489. Online ahead of print.ABSTRACTCurrent disease-modifying therapies for Huntington disease (HD) focus on lowering mutant HTT (huntingtin; mHTT) levels, and the immunosuppressant drug rapamycin is an intriguing therapeutic for aging and neurological disorders. Rapamycin interacts with FKBP1A/FKBP12 and FKBP5/FKBP51, inhibiting the MTORC1 complex and increasing cellular clearance mechanisms. Whether the levels of FKBP (FK506 binding protein) family members are altered in HD models and if these proteins are potential therapeutic targets for HD have not been invest...
Source: Autophagy - May 24, 2021 Category: Cytology Authors: Barbara J Bailus Stephen M Scheeler Jesse Simons Maria A Sanchez Kizito-Tshitoko Tshilenge Jordi Creus-Muncunill Swati Naphade Alejandro Lopez-Ramirez Ningzhe Zhang Kuruwitage Lakshika Madushani Stanislav Moroz Ashley Loureiro Katherine H Schreiber Felix Source Type: research

GSE162349 mRNA Sequencing of control and huntington's disease iPSC-derived medium spiny neuron-like cells and Q175 HET or WT mice. Plus and minus knockdown of PIAS1.
Contributors : Ryan G Lim ; Jie Wu ; Leslie M ThompsonSeries Type : Expression profiling by high throughput sequencingOrganism : Homo sapiens ; Mus musculusHD and control patient-derived induced pluripotent stem cells were used to generate medium spiny neuron-like cells. four control in duplicate and three HD samples in duplicate with CAG repeat length in juvenile onset range were differentiated as biological growth replicates (separate differentiations) into medium spiny neuron-like cells. Cells were treated with LNPs with siRNA for knockdown of PIAS1 or a luciferase control. Total RNA was isolated using the Qiagen RNeasy...
Source: GEO: Gene Expression Omnibus - January 1, 2021 Category: Genetics & Stem Cells Tags: Expression profiling by high throughput sequencing Homo sapiens Mus musculus Source Type: research

Polyglutamine Disease Modeling: Epitope Based Screen for Homologous Recombination using CRISPR/Cas9 System
Discussion We show that CRISPR/Cas9 system can be utilized to perform homologous recombination in human cells to generate a HD isogenic allelic series (21, 72, 97 CAG). In our study, we compared the Cas9 vs. Cas9 D10A enzymes for their ability to mediate this event using two distinct endpoints. The use of Cas9 D10A is likely to be more selective with less off target effects. When we compared CRISPR-assisted HR using methylene blue stained clonal colonies it appeared that the wildtype Cas9 was much more effective at mediating HR. However, when we analyzed the same experiment in our 1C2 western blot assay which only detects ...
Source: PLOS Currents Huntington Disease - April 15, 2014 Category: Neurology Authors: mahrucan Source Type: research