As a scientist involved in gene therapy, you have been asked to develop gene the
ID: 95213 • Letter: A
Question
As a scientist involved in gene therapy, you have been asked to develop gene therapy vectors based on one of these viruses to treat Parkinson’s disease and other diseases of the nervous system.
Which virus would you use as a gene therapy vector? Clearly explain why you picked this virus based on what you know about the replication cycle of the virus.
How would you design this vector? Think about where in the genome of the virus would you insert the transgene to be delivered to the nervous system and why?
Explanation / Answer
*The virus that should be used in the gene therapy for Parkinson's disease is a 'lentivirus' of retrovirus group. One of the best known example for retrovirus is 'HIV'. So, we have to take a virus as a vector which is a close relative of HIV.
*We have picked this virus because these viruses can integrate viral RNA into DNA of the host cell and can efficiently infect the non dividing cells like neurons also.As parkinson's diesease is a neural disorder and the gene therapy is to be done to nervous tissues,we have selected this virus particularly.
*The virus initially produce DNA from RNA by reverse transcription with the help of an enzyme called Reverse transcriptase.Then integrease is used to incorporate viral DNA with Host cell DNA.
*As lentivirus offer stability of transgene expression (the ability to reach the nuclei of target cells)
*The design of this vector is done in a safety way that they should exihibit seperate expression of the viral genes is trans to prevent recombination events.
*The viral genome should be replaced with 'dopamine generating genes' mainly at three regions,
The 'gag'domain - which codes for structural components of the viruses capsid,the matrix,nucleoprotiens.
The 'pol' domain - which codes for the reverse transcriptase
and integrate enzymes.
The 'env'domain - which encodes for the glycoprotiens and envelop on the surface of virous.
*It is to be delivered to nervous system(striatum part of the brain)because that is region of dopamine generation.
*And finally if the levels of dopamine is increased,parkinson's disease will be cured.
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