Results 51 to 60 of about 28,868 (258)

Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing.

Molecular Therapy, 2016
Delivery represents a significant barrier to the clinical advancement of oligonucleotide therapeutics for the treatment of neurological disorders, such as Huntington's disease.
Marie-Cécile Didiot, L. M. Hall, Andrew H Coles, R. Haraszti, B. Godinho, K. Chase, E. Sapp, Socheata Ly, J. Alterman, M. Hassler, Dimas Echeverria, Lakshmi Raj, D. V. Morrissey, M. Difiglia, N. Aronin, A. Khvorova   +15 more
semanticscholar   +1 more source

Mutant huntingtin and mitochondrial dysfunction [PDF]

Trends in Neurosciences, 2008
Huntington's disease (HD) is a fatal, inherited neurodegenerative disorder that gradually robs affected individuals of memory, cognitive skills and normal movements. Although research has identified a single faulty gene, the huntingtin gene, as the cause of the disease, a cure remains elusive.
Bossy-Wetzel, Ella, Petrilli, Alejandra, Knott, Andrew B.   +2 more
openaire   +3 more sources

The emerging role of the first 17 amino acids of huntingtin in Huntington’s disease

Biomolecular Concepts, 2015
Huntington’s disease (HD) is caused by a polyglutamine (polyQ) domain that is expanded beyond a critical threshold near the N-terminus of the huntingtin (htt) protein, directly leading to htt aggregation. While full-length htt is a large (on the order of
Arndt James R., Chaibva Maxmore, Legleiter Justin   +2 more
doaj   +1 more source

Mutant huntingtin impairs PNKP and ATXN3, disrupting DNA repair and transcription

eLife, 2019
How huntingtin (HTT) triggers neurotoxicity in Huntington’s disease (HD) remains unclear. We report that HTT forms a transcription-coupled DNA repair (TCR) complex with RNA polymerase II subunit A (POLR2A), ataxin-3, the DNA repair enzyme polynucleotide ...
Rui Gao, Anirban Chakraborty, Charlene Geater, Subrata Pradhan, K. L. Gordon, J. Snowden, Subo Yuan, A. Dickey, S. Choudhary, T. Ashizawa, L. Ellerby, A. L. La Spada, L. Thompson, T. Hazra, P. Sarkar   +14 more
semanticscholar   +1 more source

The Predicted Structure of the Headpiece of the Huntingtin Protein and Its Implications on Huntingtin Aggregation [PDF]

Journal of Molecular Biology, 2009
We have performed simulated tempering molecular dynamics simulations to study the thermodynamics of the headpiece of the Huntingtin (Htt) protein (N17(Htt)). With converged sampling, we found this peptide is highly helical, as previously proposed. Interestingly, this peptide is also found to adopt two different and seemingly stable states.
Kelley, Nicholas W., Huang, Xuhui, Tam, Stephen, Spiess, Christoph, Frydman, Judith, Pande, Vijay S.   +5 more
openaire   +4 more sources

High-content chemical and RNAi screens for suppressors of neurotoxicity in a Huntington's disease model. [PDF]

PLoS ONE, 2011
To identify Huntington's Disease therapeutics, we conducted high-content small molecule and RNAi suppressor screens using a Drosophila primary neural culture Huntingtin model.
Joost Schulte, Katharine J Sepp, Chaohong Wu, Pengyu Hong, J Troy Littleton   +4 more
doaj   +1 more source

Mutant huntingtin disrupts mitochondrial proteostasis by interacting with TIM23

Proceedings of the National Academy of Sciences of the United States of America, 2019
Significance We delineate the downstream pathologic consequences underlying the known mitochondrial protein import defect caused by mutant huntingtin (mHTT).
S. Yablonska, V. Ganesan, Lisa M. Ferrando, Jinho Kim, Anna M. Pyzel, Oxana V. Baranova, Nicolas K. Khattar, Timothy M. Larkin, S. Baranov, N. Chen, Colleen E. Strohlein, D. A. Stevens, Xiaomin Wang, Yue-Fang Chang, M. Schurdak, D. Carlisle, J. Minden, R. Friedlander   +17 more
semanticscholar   +1 more source

Taking a Break From Huntingtin [PDF]

Molecular Therapy - Nucleic Acids, 2012
Three groups have forged an important collaboration to test the utility of antisense oligonucleotides (ASOs) as a therapeutic modality for the treatment of Huntington's disease (HD), a fatal neurodegenerative disease manifesting in ~75,000 individuals in the United States and Europe alone.1 HD is one of a group of dominantly inherited neurodegenerative
openaire   +6 more sources

Rapamycin reduces neuronal mutant huntingtin aggregation and ameliorates locomotor performance in Drosophila

Frontiers in Aging Neuroscience, 2023
Huntington’s disease (HD) is a neurodegenerative disease characterized by movement and cognitive dysfunction. HD is caused by a CAG expansion in exon 1 of the HTT gene that leads to a polyglutamine (PQ) repeat in the huntingtin protein, which aggregates ...
Jonathan R. Roth, Jonathan R. Roth, Ruan Carlos Macedo de Moraes, Brittney P. Xu, Savannah R. Crawley, Malghalara A. Khan, Girish C. Melkani   +6 more
doaj   +1 more source

Purification tags markedly affect self‐aggregation of CPEB3

FEBS Letters, EarlyView.
Although recombinant proteins are used to study protein aggregation in vitro, uncleaved tags can interfere with accurate interpretation. Our findings demonstrate that His₆‐GFP and His₁₂ tags significantly affect liquid droplet and amyloid fibril formation in the intrinsically disordered region (IDR) of mouse cytoplasmic polyadenylation element‐binding ...
Harunobu Saito, Yujin Lee, Motoharu Ueno, Naotaka Sekiyama, Masatomo So, Ayako Furukawa, Kenji Sugase   +6 more
wiley   +1 more source