Suggest two reasons why people had the drug injected into the cerebrospinal fluid (lines 12–13) rather than taking a pill containing the drug

Human Biology (MindTap Course List)
11th Edition
ISBN:9781305112100
Author:Cecie Starr, Beverly McMillan
Publisher:Cecie Starr, Beverly McMillan
Chapter22: Genes And Disease: Cancer
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Question: Suggest two reasons why people had the drug injected into the cerebrospinal fluid (lines 12–13) rather than taking a pill containing the drug.
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 example numbness, muscular weakness and muscular
paralysis. Sometimes, neurones of the autonomic nervous system are
affected, causing heart rate irregularities.
Huntington's disease is a disorder caused when a protein called huntingtin
damages the brain. Huntingtin is produced because of a dominant, mutant
allele.
The first successful drug trial to reduce concentrations of huntingtin in the
human brain involved 46 patients. The patients received the drug for 4
months. The concentration of huntingtin was reduced in all the patients.
The drug was injected at the base of the spine into the cerebrospinal fluid
bathing the brain and spinal cord. The drug contains single-stranded DNA
molecules. These single-stranded molecules inhibit the MRNA needed to
produce huntingtin.
10
15
Symptoms of Huntington's disease can start at any time, but usually develop
between 30 and 50 years of age. The likelihood and age when symptoms start
are linked to the number of CAG base sequence repeats in the gene for
Huntington's disease. However, recent studies have suggested that
epigenetics may also affect the age when symptoms first start.
20
Transcribed Image Text: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 example numbness, muscular weakness and muscular paralysis. Sometimes, neurones of the autonomic nervous system are affected, causing heart rate irregularities. Huntington's disease is a disorder caused when a protein called huntingtin damages the brain. Huntingtin is produced because of a dominant, mutant allele. The first successful drug trial to reduce concentrations of huntingtin in the human brain involved 46 patients. The patients received the drug for 4 months. The concentration of huntingtin was reduced in all the patients. The drug was injected at the base of the spine into the cerebrospinal fluid bathing the brain and spinal cord. The drug contains single-stranded DNA molecules. These single-stranded molecules inhibit the MRNA needed to produce huntingtin. 10 15 Symptoms of Huntington's disease can start at any time, but usually develop between 30 and 50 years of age. The likelihood and age when symptoms start are linked to the number of CAG base sequence repeats in the gene for Huntington's disease. However, recent studies have suggested that epigenetics may also affect the age when symptoms first start. 20
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