Drugging unconventional targets: insights from Huntington's disease

Highlights • Lowering protein levels provides an alternative approach for unconventional targets. • Interferon–NUB1–KLHL22 may target mutant HTT for proteasomal degradation. • New screenings for compound modifiers of mutant HTT mRNA levels are desired.

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Bibliographic Details
Published inTrends in pharmacological sciences (Regular ed.) Vol. 35; no. 2; pp. 53 - 62
Main Authors Yu, Shenliang, Liang, Yijian, Palacino, James, Difiglia, Marian, Lu, Boxun
Format Journal Article
LanguageEnglish
Published England Elsevier Ltd 01.02.2014
Subjects
Advanced Basic Science
Animals
antisense oligonucleotides
high-throughput screening
Humans
Huntingtin Protein
Huntington Disease - drug therapy
Huntington Disease - genetics
Huntington Disease - metabolism
Molecular Targeted Therapy
Nerve Tissue Proteins - antagonists & inhibitors
Nerve Tissue Proteins - genetics
Nerve Tissue Proteins - metabolism
neurodegenerative disorder
protein degradation
proteinopathies
RNA interference
neurodegenerative disorder
RNA interference
proteinopathies
protein degradation
antisense oligonucleotides
high-throughput screening
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Summary:Highlights • Lowering protein levels provides an alternative approach for unconventional targets. • Interferon–NUB1–KLHL22 may target mutant HTT for proteasomal degradation. • New screenings for compound modifiers of mutant HTT mRNA levels are desired.
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ISSN:0165-6147
1873-3735
DOI:10.1016/j.tips.2013.12.001