The Ultimate Gene Therapy Quiz

Gene therapy is a way of altering the genes in a person's body to try to cure or stop a disease.

One approach is to produce the right transcript for a protein to function correctly. Another approach is to knock down or reduce the amount of a gene product using small interfering RNA.

One way is through virus-mediated DNA transfer, which involves infecting cells specifically with viral nucleic acid. Another way is through non-viral DNA transfer into bacteria, non-animal eukaryotic cells, and plant cells.

<p>The technique of transformation refers to a series of events resulting in a permanent change in an animal cell's phenotype and implies genetic instability and a progression to a cancerous state.</p> Signup and view all the answers

<p>Gene therapy has applications in gene function and regulation, mutational analysis and characterization, effects of gene expression on cell health, large-scale production of proteins, and stem cell engineering. It can also be used for in vivo knock-down and knock-in experiments, phenotype analysis, function recovery, pathway analysis, and drug target discovery.</p> Signup and view all the answers

<p>Virus-mediated DNA transfer involves infecting cells specifically with viral nucleic acid, which can result in a higher efficiency of gene transfer but may also have safety concerns. Non-viral DNA transfer into bacteria, non-animal eukaryotic cells, and plant cells can be safer but may have lower efficiency of gene transfer.</p> Signup and view all the answers

<p>Transformation refers to a series of events resulting in a permanent change in an animal cell's phenotype, which can lead to genetic instability and a progression to a cancerous state. This highlights the potential risks of gene therapy, as the introduction of foreign nucleic acids could potentially lead to unintended genetic changes and cellular abnormalities.</p> Signup and view all the answers

<p>Ethical considerations surrounding gene therapy include concerns about the use of viral vectors, which can have safety risks and potential side effects, as well as the possibility of germline gene therapy, which raises questions about the heritability of genetic modifications and potential unintended consequences for future generations.</p> Signup and view all the answers