[29] Advanced Gene Delivery Systems

Question 1

What are Gene Delivery Systems?

Answer 1

Methods used to introduce foreign DNA into host cells.

Question 2

What is the purpose of Gene Delivery Systems?

Answer 2

To alter the genetic makeup of cells for research, medicine, or biotechnology.

Question 3

What are examples of Advanced Gene Delivery Systems?

Answer 3

• Viral vectors (e.g., lentivirus, adenovirus)
• Non-viral vectors (e.g., liposomes, nanoparticles)

Question 4

How does a Viral Vector work in gene delivery?

Answer 4

It is modified to carry therapeutic genes instead of its viral genome and then used to infect target cells.

Question 5

What is a Non-viral Vector?

Answer 5

It includes physical methods (e.g., electroporation) and chemical methods (e.g., liposomes) to deliver genes into cells.

Question 6

How does Electroporation work in gene delivery?

Answer 6

By applying an electric field to cells, it increases the permeability of the cell membrane, allowing DNA to enter.

Question 7

How does a Liposome work in gene delivery?

Answer 7

Liposomes can encapsulate DNA and deliver it to cells by fusing with the cell membrane.

Question 8

What is the advantage of using Viral Vectors in gene delivery?

Answer 8

High efficiency of gene delivery and long-term expression of the gene.

Question 9

What is the disadvantage of using Viral Vectors in gene delivery?

Answer 9

• Potential for immune response
• Limited packaging capacity

Question 10

What is the advantage of using Non-viral Vectors in gene delivery?

Answer 10

• No viral proteins, thus less immunogenic
• Can carry larger genes

Question 11

What is the disadvantage of using Non-viral Vectors in gene delivery?

Answer 11

• Lower efficiency of gene delivery
• Transient expression of the gene

Question 12

What is the purpose of Surface Modification in gene delivery systems?

Answer 12

To target specific cells and to enhance the stability and delivery efficiency of vectors.

Question 13

What is “Gene Therapy”?

Answer 13

A technique that uses genes to treat or prevent disease.

Question 14

What role do Advanced Gene Delivery Systems play in Gene Therapy?

Answer 14

They deliver therapeutic genes to patient’s cells, forming the basis of gene therapy.

Question 15

What are the challenges in designing Advanced Gene Delivery Systems?

Answer 15

• Achieving targeted delivery
• Avoiding immune responses
• Ensuring safety and efficacy

Question 16

What is the importance of “transfection efficiency” in gene delivery systems?

Answer 16

It refers to the percentage of cells that take up and express the delivered gene, and higher efficiency leads to better experimental or therapeutic outcomes.

Question 17

How does “nanoparticle” work in gene delivery?

Answer 17

Nanoparticles can be designed to package and protect DNA, and facilitate its entry into cells.

Question 18

What are “dendrimers” in the context of gene delivery?

Answer 18

Dendrimers are highly branched molecules that can bind to DNA and deliver it into cells.

Question 19

What is “gene silencing” in the context of gene delivery?

Answer 19

It refers to techniques (like RNA interference) that inhibit the expression of specific genes. Advanced gene delivery systems can be used to deliver these inhibitory molecules.

Question 20

What are safety considerations in designing gene delivery systems?

Answer 20

• Minimizing off-target effects
• Avoiding insertional mutagenesis
• Minimizing immune responses