Gene Transfer Methods

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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?

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

What is an example of an autosomal recessive condition?

Cystic fibrosis (A) Signup and view all the answers

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

Brain and nerve damage (B) Signup and view all the answers

What is the purpose of using recombinant DNA technology?

To correct genetic defects (A) Signup and view all the answers

What is the result of physical approaches on cell membranes?

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

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

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

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

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

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

DNA enters the cells by diffusion (D) Signup and view all the answers

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

Rendering the virus replication-incompetent (D) Signup and view all the answers

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

Their genetic material (A) Signup and view all the answers

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

Liver, skin, pancreas, spleen, and tumors (B) Signup and view all the answers

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

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

What is a characteristic of adenoviral vectors?

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

Why are viruses being considered as vehicles for gene therapy?

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

What is a limitation of first-generation adenovectors?

They elicit strong immune and inflammatory responses (A) Signup and view all the answers

What has been done to improve adenoviral vectors?

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

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

Removing the genes that cause disease (A) Signup and view all the answers

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

It remains in the extrachromosomal form (A) Signup and view all the answers

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

To produce insulin in diabetics (C) Signup and view all the answers

Why do adenoviral vectors mediate short-term gene expression?

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

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

They elicit strong immune and inflammatory responses (C) Signup and view all the answers

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

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

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

Cancer and other diseases (A) Signup and view all the answers

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

CFTR (A) Signup and view all the answers

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

All of the above (D) Signup and view all the answers

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

Parkinson's disease (D) Signup and view all the answers

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

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