Gene Transfer Methods

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Questions and Answers

What is the main goal of the eugenic approach?

  • To alter or improve complex traits of a person (correct)
  • To treat recessive disorders
  • To cure autosomal dominant conditions
  • To replace a defective gene with a normal one

What is the primary limitation of replacement therapy?

  • It is not suitable for gene modification
  • It is not suitable for dominant disorders (correct)
  • It is not suitable for recessive disorders
  • It is not suitable for autosomal recessive conditions

What is the main difference between autosomal dominant and recessive conditions?

  • The number of genes required to cause the condition (correct)
  • The type of protein produced
  • The severity of the condition
  • The age of onset

What is the purpose of corrective gene therapy?

<p>To replace a defective gene with a normal one (B)</p> Signup and view all the answers

What is an example of an autosomal recessive condition?

<p>Cystic fibrosis (A)</p> Signup and view all the answers

What is the result of not being able to metabolize phenylalanine?

<p>Brain and nerve damage (B)</p> Signup and view all the answers

What is the purpose of using recombinant DNA technology?

<p>To correct genetic defects (A)</p> Signup and view all the answers

What is the result of physical approaches on cell membranes?

<p>Transient injuries or defects on the cell membrane (B)</p> Signup and view all the answers

What is the mechanism of gene delivery using a gene gun?

<p>DNA is expelled onto cells or tissue using pressurized gas (B)</p> Signup and view all the answers

What is a limitation of using a gene gun for gene transfer?

<p>It can only be used for gene transfer to skin, mucosa, or surgically exposed tissues within a confined area (D)</p> Signup and view all the answers

What is the result of DNA penetration into cells using physical approaches?

<p>DNA enters the cells by diffusion (D)</p> Signup and view all the answers

What is a minimum requirement to modify a virus for gene therapy?

<p>Rendering the virus replication-incompetent (D)</p> Signup and view all the answers

What do viruses introduce into the host cell during their replication cycle?

<p>Their genetic material (A)</p> Signup and view all the answers

What organs or tissues have been successfully targeted using gene gun technology?

<p>Liver, skin, pancreas, spleen, and tumors (B)</p> Signup and view all the answers

What happens to the genes of a virus that physically inserts its genes into the host's genome?

<p>They are incorporated into the host's genome (C)</p> Signup and view all the answers

What is a characteristic of adenoviral vectors?

<p>They can produce a high level of therapeutic protein (B)</p> Signup and view all the answers

Why are viruses being considered as vehicles for gene therapy?

<p>Because they can carry 'good' genes into a human cell (D)</p> Signup and view all the answers

What is a limitation of first-generation adenovectors?

<p>They elicit strong immune and inflammatory responses (A)</p> Signup and view all the answers

What has been done to improve adenoviral vectors?

<p>Deletion of E1, E2, and E4 genes (C)</p> Signup and view all the answers

What is the first step in modifying a virus for gene therapy?

<p>Removing the genes that cause disease (A)</p> Signup and view all the answers

What happens to the adenoviral vector genome after being released from endosomes?

<p>It remains in the extrachromosomal form (A)</p> Signup and view all the answers

What is the goal of replacing viral genes with desired genes?

<p>To produce insulin in diabetics (C)</p> Signup and view all the answers

Why do adenoviral vectors mediate short-term gene expression?

<p>Because they are recognized by the host immune system (D)</p> Signup and view all the answers

What is a challenge in using adenoviral vectors for gene therapy?

<p>They elicit strong immune and inflammatory responses (C)</p> Signup and view all the answers

What is the main characteristic of the second generation adenoviral vector?

<p>All the viral genes have been deleted from the vector (C)</p> Signup and view all the answers

What type of disease has been treated with adenoviral-based gene transfer?

<p>Cancer and other diseases (A)</p> Signup and view all the answers

What is the name of the gene transferred using adenoviral vectors for the treatment of cystic fibrosis?

<p>CFTR (A)</p> Signup and view all the answers

Which of the following diseases has been targeted using adenoviral vectors for gene therapy?

<p>All of the above (D)</p> Signup and view all the answers

What is the name of the neurodegenerative disease that has been targeted using adenoviral vectors for gene therapy?

<p>Parkinson's disease (D)</p> Signup and view all the answers

What is the advantage of using adenoviral vectors for gene therapy?

<p>They can mediate efficient gene transfer to hepatocytes (D)</p> Signup and view all the answers

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