How can genetic disorders be treated through gene therapy?
Placing a working copy of a gene into cells that only contain dysfunctional copies of the gene, transcription of the added gene will mean no longer have symptoms.
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Describe gene therapy by adding genes (augmentation)
Engineering a functioning copy of the gene into the relevant specialised cell means the polypeptide is synthesised and the cells can function normally.
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Describe gene therapy by killing specific cells
Cancers treated this way. Make cancerous cells express genes to produce proteins that make the cells vulnerable to attack by the immune system.
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What are germline cells?
Cells that can become specialised and could become a new being.
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How does germline therapy work?
Engineering a gene into sperm, an egg or a zygote means as the organism grows, every cell contains a copy of the engineered gene.
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What is the difference between germline cell gene therapy and somatic cell gene therapy?
In somatic gene cell therapy, the genetic modification is restricted to somatic body cells, with no effect on the germline.
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Why is germline gene therapy illegal?
Modification of DNA could create a new human disease or interfere with human evolution. Permanent modifications raise difficult moral, ethical and social issues.
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How is the functioning allele of the gene introduced in somatic cell gene therapy?
Functioning allele introduced into target cells so techniques to get the gene to the target location are needed.
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Is somatic cell gene therapy short or long lived and what are the issues surrounding this?
Somatic cell gene therapy is short lived so has to be repeated regularly. The cells containing the gene will NOT pass on the allele.
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What are the difficulties of getting the allele into getting the allele into the genome in a functioning state?
Genetically modified viruses have been tried but the host become immune to them and liposomes are used but these may be inefficient.
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Can the genetic manipulations be passed on to children in somatic cell gene therapy?
No, they are restricted to the actual patient.
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Where is the functioning allele of the gene introduced into?
Functioning allele introduced into germline cells - more straightforward.
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Do the offspring contain the allele in germline cell gene therapy?
Yes, the allele is passed down to the offspring.
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Other cards in this set
Card 2
Front
How can genetic disorders be treated through gene therapy?
Back
Placing a working copy of a gene into cells that only contain dysfunctional copies of the gene, transcription of the added gene will mean no longer have symptoms.
Card 3
Front
Describe gene therapy by adding genes (augmentation)
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