gene therapy + cell replacement therapy

Question 1

what is the aim of gene therapy?

Answer 1

aims to treat or cure genetic abnormalities by identifying faulty genes + inserting healthy ones

Question 2

how does gene therapy achieve its aim?

Answer 2

• replacing a mutated gene with a healthy copy
• fixing or inactivating mutated genes
• inserting a new gene that will fight the disease
• making their immune system recognize diseased cells

Question 3

describe gene therapy

Answer 3

• uses genes themselves as treatments
• focus on single-gene disorders
• corrects the underlying cause
• vector is used to deliver desired DNA into cells
• DNA is then incorporated into the nucleus of the cell and undergoes transcription + translation to produce the desired protein

Question 4

what is the aim of cell replacement therapy?

Answer 4

to introduce new, healthy cells into a patient’s body, to replace the diseased or missing ones.

Question 5

what are stem cells?

Answer 5

• undifferentiated cells that are capable of repeated mitotic divisions for long periods of time + differentiate into specialized cells

Question 6

describe cell replacement therapy

Answer 6

• replacing dying neurons with healthy neural tissue
• used on disorders involving loss of or injury to normal cells
• cells survive + grow connections with neurons

Question 7

compare cell therapy and gene therapy

Answer 7

gene therapy

• aims to treat diseases by replacing, inactivating or introducing genes into cells
• can be in vivo or ex vivo

cell therapy

• aims to treat diseases by restoring or altering certain sets of cells or by using cells to carry a therapy through the body
• cells are cultivated out of the body before being injected into the patient
• can originate from the patient of a donor

Question 8

describe the process of gene therapy

Answer 8

(isolate a gene, and cut with restriction enzymes to create a recombinant plasmid)

1) the plasmid inserted into the vector
2) introduced to cell culture
3) vector taken in by cell + packed into the vesicle
4) move to nucleus + vesicle broken down
5) gene integrated w the patient’s DNA
6) cells are now functional as they have the DNA

Question 9

how can cystic fibrosis use gene therapy?

Answer 9

• lung = easy access
• therapy can begin before significant lung damage
• modified common cold virus to act as a vector to carry normal genes to CF cells in the airway of the lungs

Question 10

how can diabetes be treated/cured by gene therapy?

Answer 10

• type 1 = beta cells are destroyed
• the body cannot produce insulin = high glucose levels
• reprogram other cells to produce insulin
  1) gene for insulin is introduced into a vector
  2) vector then used to infect desired cells (alpha cells)
  3) cells incorporate new DNA into the nucleus
  4) use protein synthesis to produce insulin

Question 11

what is the process of cell replacement therapy?

Answer 11

1) cell wanted is grown on a scaffold of material
2) scaffold is then surgically placed into the body
3) cells then replicate
3) scaffold is absorbed by cells and breaks down

Question 12

what is a scaffold in cell replacement therapy?

Answer 12

• natural/synthetic material where cells are grown
• template for tissue growth
• large pore size = cells can grow and discussion of nutrients
• biodegradable = absorbed by cells not removed