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Condition: Huntington's Disease

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Total 63 results found since Jan 2013.

A Feedback Loop between TGF- β1 and < em > ATG5 < /em > Mediated by miR-122-5p Regulates Fibrosis and EMT in Human Trabecular Meshwork Cells
Curr Issues Mol Biol. 2023 Mar 13;45(3):2381-2392. doi: 10.3390/cimb45030154.ABSTRACTAutophagy is a cell's evolutionary conserved process for degrading and recycling cellular proteins and removing damaged organelles. There has been an increasing interest in identifying the basic cellular mechanism of autophagy and its implications in health and illness during the last decade. Many proteinopathies such as Alzheimer's and Huntington's disease are reported to be associated with impaired autophagy. The functional significance of autophagy in exfoliation syndrome/exfoliation glaucoma (XFS/XFG), remains unknown though it is pres...
Source: Current Issues in Molecular Biology - March 28, 2023 Category: Molecular Biology Authors: Munmun Chakraborthy Aparna Rao Source Type: research

CRISPR-Cas9 mediated genome editing of Huntington's disease neurospheres
CONCLUSION: Our study confirmed that CAG repeat of R6/2 mouse-derived neurospheres can be edited through CRISPR-Cas9. Editing of CAG repeat sequence decreases polyglutamine aggregation and cellular apoptosis of HD neurospheres, which may be related to the increased expressions of PGC-1α and BDNF. Our data provide the evidence that CRISPR-Cas9 mediated genome editing has therapeutic potential on HD neuronal cells.PMID:36550260 | DOI:10.1007/s11033-022-08175-6
Source: Mol Biol Cell - December 22, 2022 Category: Molecular Biology Authors: Ji Yun Han Jaewoo Seo Yoori Choi Wooseok Im Jae-Jun Ban Jung-Joon Sung Source Type: research

Modified cyclodextrin-based nanoparticles mediated delivery of siRNA for Huntingtin gene silencing across an in vitro BBB model
In conclusion, the CD platform is a promising option for delivery of siRNA-based therapeutics for HD with wider potential to treat other diseases with a genetically validated target in the central nervous system.PMID:34793942 | DOI:10.1016/j.ejpb.2021.11.003
Source: European Journal of Pharmaceutics and Biopharmaceutics - November 18, 2021 Category: Drugs & Pharmacology Authors: Monique C P Mendon ça Michael F Cronin John F Cryan Caitriona M O'Driscoll Source Type: research

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

Enriched chitosan nanoparticles loaded with siRNA are effective in lowering Huntington's disease gene expression following intranasal administration
Publication date: Available online 27 October 2019Source: Nanomedicine: Nanotechnology, Biology and MedicineAuthor(s): Vasyl Sava, Oksana Fihurka, Anastasia Khvorova, Juan Sanchez-RamosAbstractTherapies to lower gene expression in brain disease currently require chronic administration into the cerebrospinal fluid (CSF) by intrathecal infusions or direct intracerebral injections. Though well-tolerated in the short-term, this approach is not tenable for a life-time of administration. Nose-to-brain delivery of enriched chitosan-based nanoparticles loaded with anti-HTT siRNA was studied in a transgenic YAC128 mouse model of Hu...
Source: Nanomedicine: Nanotechnology, Biology and Medicine - October 28, 2019 Category: Nanotechnology Source Type: research

Loss of huntingtin function slows synaptic vesicle endocytosis in striatal neurons from the httQ140/Q140 mouse model of Huntington's disease.
Abstract Huntington's disease (HD) is caused by CAG repeat expansion within the HTT gene, with the dysfunction and eventual loss of striatal medium spiny neurons a notable feature. Since medium spiny neurons receive high amounts of synaptic input, we hypothesised that this vulnerability originates from an inability to sustain presynaptic performance during intense neuronal activity. To test this hypothesis, primary cultures of either hippocampal or striatal neurons were prepared from either wild-type mice or a knock-in HD mouse model which contains 140 poly-glutamine repeats in the huntingtin protein (httQ140/Q140...
Source: Neurobiology of Disease - October 11, 2019 Category: Neurology Authors: McAdam RL, Morton A, Gordon SL, Alterman JF, Khvorova A, Cousin MA, Smillie KJ Tags: Neurobiol Dis Source Type: research

Assessing Triplet Repeat Expansions in Human SVG-A Cell Culture.
Abstract Determining the molecular mechanisms that contribute to trinucleotide repeat (TNR) expansions is essential to understanding the origin of genetically inherited diseases, such as Huntington's disease, and to inform efforts in developing therapeutic treatments. As one resource to probe the mechanisms of TNR expansions, we describe an expansion assay in human tissue culture cells. The cell line SVG-A, derived from human astrocytes, has the important property of supporting expansions in culture, unlike many cell lines derived from patients. SVG-A cells are also amenable to standard genetic and biochemical tec...
Source: Mol Biol Cell - October 7, 2019 Category: Molecular Biology Authors: Williams GM, Lahue RS Tags: Methods Mol Biol Source Type: research

Current Development of siRNA Bioconjugates: From Research to the Clinic
In this study, it was shown that the main factor determining the nature of the biodistribution of conjugates is their lipophilicity. Conjugates of siRNA with lower lipophilicity; i.e., derivatives of retinoic acid, lithocholic acid, and docosahexanoic acid with greater efficiency than cholesterol conjugates accumulated in the kidneys, bladder, and lungs of the mouse after subcutaneous injection (Biscans et al., 2018). This fact is consistent with previous data that showed that more lipophilic conjugates bind more efficiently to serum components, and thus are not excreted by the kidneys (Wolfrum et al., 2007; Osborn et al.,...
Source: Frontiers in Pharmacology - April 25, 2019 Category: Drugs & Pharmacology Source Type: research