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Guillain–Barré syndrome is a rare disease in which the immune system damages the myelin sheath of neurones. Myelin sheath damage can cause a range of symptoms, for e... show full transcript
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The damage to the myelin sheath results in impaired transmission of nerve impulses along the affected neurones. This occurs because the myelin sheath facilitates saltatory conduction, where the impulse jumps between the nodes of Ranvier. Without proper myelination, the conduction is slowed or disrupted, leading to insufficient stimulation of the muscles, resulting in paralysis.
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Guillain–Barré syndrome can affect the autonomic nervous system, which regulates involuntary functions including heart rate. Damage to the myelin sheath can disrupt the conduction of nerve impulses to the cardiac muscle, resulting in irregular heart rhythms due to improper electrical signaling.
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The single-stranded DNA molecules are complementary to the mRNA transcribed from the huntingtin gene. When injected, they bind to the mRNA, preventing its translation into the huntingtin protein. This reduces the overall concentration of huntingtin in the brain, helping to alleviate some symptoms of Huntington's disease.
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Firstly, the drug only reduces levels of the huntingtin protein but does not address any underlying genetic mutation causing the disease. Secondly, the damage already done to the brain may be irreversible, meaning that while the drug can lower protein concentrations, it cannot repair any existing neurological damage.
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Injecting the drug into the cerebrospinal fluid allows for direct delivery to the central nervous system, bypassing the blood-brain barrier, which can limit drug efficacy if taken orally. Additionally, delivering the drug via injection can achieve faster and more concentrated therapeutic levels within the brain.
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Epigenetics involves changes in gene expression without alterations to the underlying DNA sequence. Modifications such as methylation of DNA can influence the expression of the huntingtin gene. If epigenetic changes lead to increased expression of the huntingtin gene in earlier years, symptoms may manifest sooner, while decreased expression may delay symptom onset.
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