Gene Therapy Worksheet by 9Q1057zw

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									                              Gene Therapy Worksheet
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One of the methods used for gene insertion in gene therapy involves using a virus. In order
that the virus cannot replicate itself once inserted into the cell and cause damage to the
host, the gene that codes for replication is removed from the virus. This is then replaced
with a sequence of DNA that contains the normal allele for the desired protein that we want
to introduce into the host. For example, in treating cystic fibrosis sufferers the DNA inserted
would encode for the normal CFTR protein. Along with this gene, a promoter sequence of
DNA which initiates transcription and translation of the inserted gene once inside the host,
would also be inserted into the viral DNA. The virus is now ready to infect the host cells and
introduce its DNA. Different types of viruses can be used – those whose DNA becomes part
of the host DNA once infected, or those whose DNA remains independent. The latter type of
virus is used when treating CF. Once the viral DNA is in the cell, the idea is that the normal
process of protein synthesis occurs producing functional proteins from the DNA that has
been inserted into the virus.



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