Huntington's disease: from molecular pathogenesis to clinical treatment

@article{Ross2011HuntingtonsDF,
  title={Huntington's disease: from molecular pathogenesis to clinical treatment},
  author={Christopher A. Ross and Sarah J. Tabrizi},
  journal={The Lancet Neurology},
  year={2011},
  volume={10},
  pages={83-98}
}
  • C. Ross, S. Tabrizi
  • Published 31 January 2011
  • Biology, Psychology, Medicine
  • The Lancet Neurology
View on Elsevier
ijdrt.com
1,259 Citations
Highly Influential Citations
72
Background Citations
575
Methods Citations
13
Results Citations
8

Figures from this paper

1,259 Citations

Citation Type

Citation Type

Huntington's disease: a clinical review
TLDR
Antisense oligonucleotide therapy is one such approach with clinical trials currently under way that may bring us one step closer to treating and potentially preventing this devastating condition.
Clinical Features of Huntington's Disease.
TLDR
The epidemiology, genetics and pathogenesis of HD as well as the clinical presentation and management of HD, which is currently focused on symptomatic treatment are discussed.
Huntington's disease: fighting on many fronts.
TLDR
Two articles in this issue of Brain highlight the Huntington's disease research community's multi-faceted approach to developing new treatments for Huntington’s disease.
Pathogenic insights from Huntington's disease-like 2 and other Huntington's disease genocopies.
TLDR
It is suggested that HDL2 is a complex disease in which transcripts and proteins expressed bidirectionally from the junctophilin-3 locus contribute to pathogenesis through both gain-and loss-of-function mechanisms.
Huntington’s Disease: Molecular Pathogenesis and New Therapeutic Perspectives
TLDR
The preclinical and clinical development molecular therapies targeting HTT expression and the modulation of biological mechanisms thought to contribute to disease pathogenesis via novel therapeutic agents are reviewed.
Neuroimmunology of Huntington's Disease: Revisiting Evidence from Human Studies
TLDR
The current review summarizes the available evidence about immune and/or inflammatory changes in HD, which is associated with increased inflammatory mediators in both the central nervous system and periphery.
Molecular Mechanisms Underlying Neuropathology in Mouse Models of Huntington’s Disease
TLDR
This dissertation will detail work performed in characterizing the cellular and molecular determinants of HD pathogenesis in novel mouse models developed in the Yang lab, using conditional genetics to define cells in the cortex and striatum as essential to developing HD phenotypes, and assessed molecular signatures in these cells that are dependent on the mutant huntingtin protein.
Huntington’s Disease: The Past, Present, and Future Search for Disease Modifiers
TLDR
Although silencing Htt expression appears promising as a disease modifying treatment, it should be attempted with caution in light of Htt’s essential roles in neural maintenance and development.
A Clinical Review on Huntington Disease
  • P. Malkani
  • Biology, Medicine
    Global Journal of Pharmacy & Pharmaceutical Sciences
  • 2018
TLDR
The mechanism by which neuronal degeneration and cell death are being generated in HD may include excitotoxicity, energy deficit, oxidative stress, inflammatory process and protein aggregation.
Preclinical and Clinical Investigations of Mood Stabilizers for Huntington's Disease: What Have We Learned?
TLDR
Preclinical and clinical investigations of the beneficial effects of lithium and VPA on HD pathophysiology are discussed, including behavioral and motor improvement, enhanced neuroprotection, and lifespan extension.
...
...

References

SHOWING 1-10 OF 250 REFERENCES
Beyond the brain: widespread pathology in Huntington's disease
Huntington's disease: Silencing a brutal killer
Selective neuronal degeneration in Huntington's disease.
Pathogenic mechanisms in Huntington's disease.
Huntington's disease: from pathology and genetics to potential therapies.
TLDR
Not all of the effects of mutant huntingtin may be cell-autonomous, and it is possible that abnormalities in neighbouring neurons and glia may also have an impact on connected cells.
Huntington's disease: phenomenological diversity of a neuropsychiatric condition that challenges traditional concepts in neurology and psychiatry.
TLDR
The following case report emphasizes the spectrum and clinical significance of neuropsychiatric phenomena associated with Huntington’s disease and discusses the diagnostic power of specific neuropsychological approaches.
Development of biomarkers for Huntington's disease
Global changes to the ubiquitin system in Huntington's disease
TLDR
A mass-spectrometry-based method is exploited to quantify polyubiquitin chains and it is demonstrated that the abundance of these chains is a faithful endogenous biomarker of UPS function, establishing that UPS dysfunction is a consistent feature of HD pathology.
Huntington's disease: the challenge for cell biologists.
Magnetic resonance imaging of Huntington's disease: preparing for clinical trials
...
...