30 Questions
What is the main goal of the eugenic approach?
To alter or improve complex traits of a person
What is the primary limitation of replacement therapy?
It is not suitable for dominant disorders
What is the main difference between autosomal dominant and recessive conditions?
The number of genes required to cause the condition
What is the purpose of corrective gene therapy?
To replace a defective gene with a normal one
What is an example of an autosomal recessive condition?
Cystic fibrosis
What is the result of not being able to metabolize phenylalanine?
Brain and nerve damage
What is the purpose of using recombinant DNA technology?
To correct genetic defects
What is the result of physical approaches on cell membranes?
Transient injuries or defects on the cell membrane
What is the mechanism of gene delivery using a gene gun?
DNA is expelled onto cells or tissue using pressurized gas
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
What is the result of DNA penetration into cells using physical approaches?
DNA enters the cells by diffusion
What is a minimum requirement to modify a virus for gene therapy?
Rendering the virus replication-incompetent
What do viruses introduce into the host cell during their replication cycle?
Their genetic material
What organs or tissues have been successfully targeted using gene gun technology?
Liver, skin, pancreas, spleen, and tumors
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
What is a characteristic of adenoviral vectors?
They can produce a high level of therapeutic protein
Why are viruses being considered as vehicles for gene therapy?
Because they can carry 'good' genes into a human cell
What is a limitation of first-generation adenovectors?
They elicit strong immune and inflammatory responses
What has been done to improve adenoviral vectors?
Deletion of E1, E2, and E4 genes
What is the first step in modifying a virus for gene therapy?
Removing the genes that cause disease
What happens to the adenoviral vector genome after being released from endosomes?
It remains in the extrachromosomal form
What is the goal of replacing viral genes with desired genes?
To produce insulin in diabetics
Why do adenoviral vectors mediate short-term gene expression?
Because they are recognized by the host immune system
What is a challenge in using adenoviral vectors for gene therapy?
They elicit strong immune and inflammatory responses
What is the main characteristic of the second generation adenoviral vector?
All the viral genes have been deleted from the vector
What type of disease has been treated with adenoviral-based gene transfer?
Cancer and other diseases
What is the name of the gene transferred using adenoviral vectors for the treatment of cystic fibrosis?
CFTR
Which of the following diseases has been targeted using adenoviral vectors for gene therapy?
All of the above
What is the name of the neurodegenerative disease that has been targeted using adenoviral vectors for gene therapy?
Parkinson's disease
What is the advantage of using adenoviral vectors for gene therapy?
They can mediate efficient gene transfer to hepatocytes
Explore the different approaches to gene transfer, including recombinant DNA technology and physical methods. Learn about the suppression of mutations and enhancement of gene transfer efficiency.
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