Results 61 to 70 of about 40,748 (225)

Therapeutic Antisense Targeting of Huntingtin [PDF]

open access: yesDNA and Cell Biology, 2020
Antisense oligonucleotides (ASOs) are a relatively new therapeutic entity that utilizes short chemically modified strands of DNA in targeted interactions with RNA to modulate the type or amount of resultant protein. This brief review summarizes the preclinical, translational, and early clinical development of an ASO designed to reduce the production of
Smith, AV, Tabrizi, SJ
openaire   +3 more sources

Huntingtin-mediated axonal transport requires arginine methylation by PRMT6

open access: yesCell Reports, 2021
Summary: The huntingtin (HTT) protein transports various organelles, including vesicles containing neurotrophic factors, from embryonic development throughout life. To better understand how HTT mediates axonal transport and why this function is disrupted
Alice Migazzi   +17 more
doaj   +1 more source

A Tac1‐Expressing Brainstem Pathway Underlies the Pathogenesis of Trigeminal Neuralgia

open access: yesAdvanced Science, EarlyView.
A critical TG‐Sp5CTac1‐PBNTac1 pathway drives trigeminal neuropathic pain (TNP). Tac1‐expressing parabrachial nucleus (PBNTac1) neurons exhibit heightened responses to innocuous stimuli in TNP, and chemogenetic inhibition of these neurons effectively prevents TNP development.
Liting Sun   +11 more
wiley   +1 more source

Raft-like microdomains play a key role in mitochondrial impairment in lymphoid cells from patients with Huntington's disease

open access: yesJournal of Lipid Research, 2012
Huntington's disease (HD) is a genetic neurodegenerative disease characterized by an exceedingly high number of contiguous glutamine residues in the translated protein, huntingtin (Htt).
Laura Ciarlo   +10 more
doaj   +1 more source

Hsp40 overexpression in pacemaker neurons delays circadian dysfunction in a Drosophila model of Huntington's disease

open access: yesDisease Models & Mechanisms, 2022
Circadian disturbances are early features of neurodegenerative diseases, including Huntington's disease (HD). Emerging evidence suggests that circadian decline feeds into neurodegenerative symptoms, exacerbating them.
Pavitra Prakash   +2 more
doaj   +1 more source

Intrabody Gene Therapy Ameliorates Motor, Cognitive, and Neuropathological Symptoms in Multiple Mouse Models of Huntington's Disease [PDF]

open access: yes, 2009
Huntington's disease (HD) is an autosomal dominant neurodegenerative disease resulting from the expansion of a glutamine repeat in the huntingtin (Htt) protein.
Ko, Jan   +2 more
core   +2 more sources

Mitochondrial Enzymes Mimetic Ultrasmall Palladium Nanozymes Prevent Senescence and Neurodegeneration Through Metabolic Reprogramming

open access: yesAdvanced Science, EarlyView.
Simply constructed polyvinylpyrrolidone (PVP) modified palladium nanoparticles (PdP NPs) as cytochrome c oxidase (CcO) and superoxide dismutase (SOD) mimic are promising multifunctional nanoreactors to boost mitochondrial respiration chain function and refine homeostasis via regulation of mitochondrial dynamics and proteostasis sensor UPRmt, which ...
Wenshu Cong   +8 more
wiley   +1 more source

Wild-Type Huntingtin Reduces the Cellular Toxicity of Mutant Huntingtin In Vivo [PDF]

open access: yesThe American Journal of Human Genetics, 2001
We have developed yeast artificial chromosome (YAC) transgenic mice expressing normal (YAC18) and mutant (YAC46 or YAC72) human huntingtin (htt), in a developmental- and tissue-specific manner, that is identical to endogenous htt. YAC72 mice develop selective degeneration of medium spiny projection neurons in the lateral striatum, similar to what is ...
Leavitt, Blair R.   +6 more
openaire   +2 more sources

PIN1 Modulates Huntingtin Levels and Aggregate Accumulation: An In vitro Model

open access: yesFrontiers in Cellular Neuroscience, 2017
Huntington's disease (HD) is a dominantly inherited neurodegenerative disorder characterized by a polyglutamine expansion within the N-terminal region of huntingtin protein (HTT).
Alisia Carnemolla   +2 more
doaj   +1 more source

Integrated genomics and proteomics define huntingtin CAG length-dependent networks in mice. [PDF]

open access: yes, 2016
To gain insight into how mutant huntingtin (mHtt) CAG repeat length modifies Huntington's disease (HD) pathogenesis, we profiled mRNA in over 600 brain and peripheral tissue samples from HD knock-in mice with increasing CAG repeat lengths.
A Dobin   +94 more
core   +1 more source

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