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Amyloid Fibrils03:03

Amyloid Fibrils

Amyloid fibrils are aggregates of misfolded proteins.  Under most circumstances, misfolded proteins are either refolded by chaperone proteins or degraded by the proteasome. However, in the case of a mutation or a disease, these proteins can accumulate to form large clusters and often further assemble to form elongated fibers, called fibrils. 
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Amyloid Fibrils03:03

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Related Experiment Video

Updated: May 31, 2026

Real-Time Fluorescent Measurement of Synaptic Functions in Models of Amyotrophic Lateral Sclerosis
08:59

Real-Time Fluorescent Measurement of Synaptic Functions in Models of Amyotrophic Lateral Sclerosis

Published on: July 16, 2021

RNA interference and amyotrophic lateral sclerosis.

Albert A Rizvanov1, Sukru Gulluoglu, Mehmet E Yalvaç

  • 1Department of Genetics, Faculty of Biology and Soil Sciences, Kazan (Volga Region) Federal University, ul. Kremlevskaya 18, R-420008 Kazan, Russia.

Current Drug Metabolism
|July 12, 2011
PubMed
Summary
This summary is machine-generated.

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease. RNA interference (RNAi) offers a promising therapeutic strategy for familial ALS by targeting gene expression, particularly in SOD1-related cases.

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Area of Science:

  • Neuroscience
  • Genetics
  • Molecular Biology

Background:

  • Amyotrophic lateral sclerosis (ALS) is a progressive motor neuron disease with unknown causes in most cases.
  • Mutations in the superoxide dismutase-1 (SOD1) gene are a known cause of familial ALS.
  • Understanding motor neuron degeneration is crucial for developing effective treatments.

Purpose of the Study:

  • To review current research on RNA interference (RNAi) as a therapeutic approach for ALS.
  • To explore the role of RNAi in gene expression regulation relevant to ALS pathogenesis.
  • To highlight the potential of RNAi for treating familial ALS, especially SOD1-related forms.

Main Methods:

  • Literature review of recent studies on RNAi and ALS.
  • Analysis of research on gene silencing mechanisms in neurodegenerative diseases.
  • Synthesis of findings on RNAi applications in preclinical and clinical ALS models.

Main Results:

  • RNA interference (RNAi) has emerged as a powerful tool for gene silencing.
  • RNAi-based strategies show potential for targeting the genetic underpinnings of familial ALS.
  • Successful application of RNAi in preclinical models suggests therapeutic viability.

Conclusions:

  • RNAi represents a significant advancement in the study and potential treatment of ALS.
  • Targeting SOD1 mutations with RNAi is a key focus for familial ALS therapy.
  • Further research into RNAi mechanisms and delivery is essential for clinical translation in ALS.