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>It is essential for function.</p> Signup and view all the answers

<p>To inhibit the expression of specific proteins.</p> Signup and view all the answers

<p>Gene function and regulation, mutational analysis and characterization, effects of gene expression on cell health, large-scale production of proteins, stem cell engineering, 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>No need for host integration, immediate expression of the gene of interest, and the ability to transfect multiple mRNAs at once.</p> Signup and view all the answers

<p>Through viral transduction to insert a working copy of the ADA gene.</p> Signup and view all the answers

<p>Magnetofection.</p> Signup and view all the answers

<p>Ethical considerations include determining what is considered normal variation versus disease, deciding whether disabilities should be cured, considering whether gene therapy demeans the lives of those with disabilities, and determining whether somatic or germ line gene therapy is more ethical. These considerations are important to address in order to ensure that gene therapy is used in a responsible and ethical manner.</p> Signup and view all the answers

<p>Examples of gene therapy techniques include transfection, transduction, and transformation. Understanding the strengths and weaknesses of these techniques is important in order to improve current gene therapy techniques and ensure their effectiveness and safety.</p> Signup and view all the answers

<p>Understanding ethical considerations in gene therapy is important in order to ensure that it is used in a responsible and ethical manner. One can become more aware of these considerations by researching and reading up on current gene therapy techniques and improvements, as well as by staying informed about ethical debates and discussions surrounding gene therapy.</p> Signup and view all the answers

<p>Knocking down gene products involves reducing the amount of a specific gene's activity, while transformation involves permanently changing the phenotype of an animal cell. Knocking down gene products can be achieved through the introduction of nucleic acids, while transformation can lead to genetic instability and a progression to a cancerous state.</p> Signup and view all the answers

<p>Producing the correct transcript for good protein function in gene therapy involves altering the genes within a person's body cells. This can be achieved through the introduction of nucleic acids into animal cells, including virus-mediated DNA transfer.</p> Signup and view all the answers

<p>Gene therapy is the process of altering the genes within a person's body cells in an effort to cure or prevent diseases. It aims to produce the correct transcript for good protein function and can be achieved through various techniques, including the introduction of nucleic acids and virus-mediated DNA transfer.</p> Signup and view all the answers

<p>Homologous recombination is the exchange of DNA strands of similar or identical nucleotide sequences. It can be used for error-free repair of double-stranded DNA breaks in gene therapy.</p> Signup and view all the answers

<p>Some factors that influence successful transfection include the ratio of DNA to transfection reagents, cell density, and degree of cell confluence.</p> Signup and view all the answers

<p>Ethical considerations for gene therapy include deciding what is normal variation and what constitutes a disease, whether disabilities should be cured, and the ethical implications of somatic gene therapy compared to germ line gene therapy. Addressing these considerations is important to ensure that gene therapy is used in a responsible and ethical manner.</p> Signup and view all the answers

<p>Scientists have used integrating targeted DNA sequences with PCR products, mixing plasmids and restriction enzymes, and applying selective pressure to improve DNA transfection. They have also used magnetic fields to focus the gene-carrying particles over the target site and enhance transfection. Virus-mediated gene therapy has shown many advantages over DNA transfection, including immediate expression of genes of interest without the need for host integration and the ability to transfect multiple mRNAs simultaneously.</p> Signup and view all the answers

<p>Virus-mediated gene therapy has shown many advantages over DNA transfection, including immediate expression of genes of interest without the need for host integration and the ability to transfect multiple mRNAs simultaneously.</p> Signup and view all the answers

<p>Magnetic fields have been used to focus the gene-carrying particles over the target site and enhance transfection efficiency by bringing the particles closer to the cells. This method has shown promising results in improving transfection efficiency.</p> Signup and view all the answers

<p>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>It is essential to study the promoter and enhancer sequences, protein: protein interactions, and knockdown of specific proteins through RNA interference or CRISPR/CAS9.</p> Signup and view all the answers

<p>To cure diseases caused by genetic mutations and polymorphisms, it is critical to understand how the body works normally and the biological etiology of diseases.</p> Signup and view all the answers

<p>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>It is crucial for effective gene therapy because the expression of the protein in eukaryotic cells determines the effectiveness of the therapy.</p> Signup and view all the answers

<p>It is critical to understand the biological etiology of diseases for effective gene therapy to cure diseases caused by genetic mutations and polymorphisms.</p> Signup and view all the answers

<p>Ethical considerations in gene therapy include concerns about whether it is ethical to change the genes of any living thing, deciding what constitutes a disease, and whether treatment should be offered to all. It is important to address these concerns to ensure that gene therapy is used responsibly and with consideration for the potential consequences.</p> Signup and view all the answers

<p>Gene therapy presents a valuable tool for studying gene expression and understanding the genetic basis of diseases. By targeting specific genes, researchers can develop new treatments and potentially cure genetic disorders.</p> Signup and view all the answers

<p>Challenges associated with gene therapy include ensuring that the correct genes are targeted, avoiding unintended consequences such as immune reactions, and determining the appropriate dosages and delivery methods for treatment.</p> Signup and view all the answers