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Questions and Answers
What is one way that ASOs can modify gene function?
What is one way that ASOs can modify gene function?
- By altering the DNA sequence directly
- By inhibiting viral replication
- By increasing protein production
- By modulating alternative splicing of RNA molecules (correct)
What is one potential use for ASOs?
What is one potential use for ASOs?
- Treating bacterial infections
- Treating neurodegenerative disorders (correct)
- Treating cancer
- Treating heart disease
What has been shown in animal models of Huntington's disease when ASOs target the mutant huntingtin gene?
What has been shown in animal models of Huntington's disease when ASOs target the mutant huntingtin gene?
- No improvement in motor function or survival
- Improvement in motor function but not survival
- Improvement in survival but not motor function
- Improvement in both motor function and survival (correct)
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Study Notes
- Antisense oligonucleotides (ASOs) are synthetic molecules that bind to complementary RNA sequences to modify gene expression and protein production.
- ASOs can be used as a therapeutic approach to target disease-causing genes and modify their function.
- ASOs can trigger gene silencing through RNase H-mediated degradation or steric blocking.
- ASOs can modulate alternative splicing of RNA molecules to generate different mRNA isoforms.
- ASOs can modify gene function through RNA editing.
- ASOs have been studied for potential use in neurodegenerative disorders, genetic disorders, and viral infections.
- ASOs targeting the mutant huntingtin gene have shown improvements in motor function and survival in animal models of Huntington's disease.
- ASOs targeting specific exonic splicing enhancers have shown promise in restoring dystrophin production in Duchenne muscular dystrophy patients.
- ASOs targeting influenza A virus RNA have been shown to inhibit viral replication.
- ASOs offer potential treatments for a wide range of diseases.
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