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Related Concept Videos

Intrinsically Disordered Proteins02:18

Intrinsically Disordered Proteins

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Intrinsically disordered proteins are a group of proteins that do not fold into specific three-dimensional structures. Their structural flexibility allows them to complement ordered proteins to perform functions that are inaccessible to rigid structures. They are more common in eukaryotes than prokaryotes and may either be exclusively intrinsically disordered or hybrid proteins, consisting of a mix of ordered and disordered regions. The absence of a rigid structure in these proteins can be...
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Aging and its effect on bone remodeling is the most common cause of bone disorders. In young and healthy people, bone deposition and resorption happen at an equal rate to maintain optimal bone health.
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Disorders of erythrocytes, or red blood cells (RBCs), include a range of conditions affecting their number, shape, or function.
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Leukocyte disorders can lead to either leukopenia, characterized by an abnormally low leukocyte count, or leukocytosis, marked by a very high leukocyte number.
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Other Disorders of Digestive System

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The gastrointestinal tract is susceptible to various disorders. If the lower esophageal sphincter is damaged, stomach acid can flow back into the esophagus, causing irritation and inflammation of the lining. This condition is called gastroesophageal reflux disease (known as heartburn) and may cause chest pain and difficulty swallowing. In the stomach, prolonged use of nonsteroidal anti-inflammatory drugs like aspirin, chronic alcohol consumption, bacterial infections such as Helicobacter...
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Panic Disorder

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Panic disorder is an anxiety disorder characterized by recurrent and sudden minutes-long episodes of intense fear, known as panic attacks. These attacks may feel like heart attacks and often happen without warning or a specific cause. They can include symptoms such as rapid heart rate, shortness of breath, chest pain, trembling, sweating, dizziness, and a sense of helplessness. During a panic attack, individuals may feel as though they are experiencing a heart attack or are in a...
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Related Experiment Video

Updated: Feb 9, 2026

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System
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Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System

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Antisense oligonucleotides in neurological disorders.

Claudia D Wurster1, Albert C Ludolph2

  • 1Department of Neurology, University of Ulm, Oberer Eselsberg 45, Ulm, 89081, Germany.

Therapeutic Advances in Neurological Disorders
|June 2, 2018
PubMed
Summary

Antisense oligonucleotides (ASOs) are revolutionizing treatment for neurodegenerative and neuromuscular diseases. These genetic therapies, approved in 2016, offer a direct strategy to modify gene expression and treat conditions like spinal muscular atrophy and Duchenne muscular dystrophy.

Keywords:
Alzheimer’s diseaseDuchenne muscular dystrophyHuntington’s diseaseamyotrophic lateral sclerosisantisense oligonucleotidesmyotonic dystrophynusinersenspinal muscular atrophy

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Area of Science:

  • Neurogenetics
  • Molecular Biology
  • Pharmacology

Background:

  • Genetics has advanced understanding of neurodegenerative and neuromuscular diseases, but treatments remain limited.
  • Antisense oligonucleotides (ASOs) represent a breakthrough, transitioning from research to clinical application.
  • Recent approvals highlight ASO efficacy in treating Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA).

Purpose of the Study:

  • To review the fundamental mechanisms of antisense oligonucleotide (ASO) action.
  • To discuss chemical modifications enhancing ASO pharmacodynamics and pharmacokinetics.
  • To explore ASO applications in treating various neurodegenerative and neuromuscular disorders.

Main Methods:

  • Review of scientific literature on ASO mechanisms and applications.
  • Analysis of ASO chemical modifications for improved therapeutic properties.
  • Focus on specific ASO-based treatments for neurological and muscular diseases.

Main Results:

  • ASOs are synthetic nucleic acid strings that modulate protein synthesis by binding to pre-mRNA/mRNA.
  • Chemical modifications are crucial for optimizing ASO efficacy and delivery.
  • ASOs show promise for treating SMA, DMD, myotonic dystrophies, Huntington's disease, ALS, and Alzheimer's disease.

Conclusions:

  • ASOs offer a direct therapeutic strategy for influencing gene expression, complementing gene replacement.
  • The development and application of ASOs represent a significant advancement in treating genetic neurological and neuromuscular disorders.
  • Further research into ASO mechanisms and modifications will expand their therapeutic potential.