Vectors gene therapy (Medical Interventions)

2.2.1 Gene Therapy- PLTW Medical Interventions

plasmid

1. Max Gene Size and Nucleic Acid Type

2. Cell Specificity

3. trigger an immune response? (Y/N)

4. does it integrate into host cell’s genome? (Y/N)

1. all sizes; DNA

2. none

3. N

4. N

liposome

1. Max Gene Size and Nucleic Acid Type

2. Cell Specificity

3. trigger an immune response? (Y/N)

4. does it integrate into host cell’s genome? (Y/N)

1. all sizes

2. none

3. N

4. N

herpes virus

1. Max Gene Size and Nucleic Acid Type

2. Cell Specificity

3. trigger an immune response? (Y/N)

4. does it integrate into host cell’s genome? (Y/N)

1. 20 kb (base pairs); DNA

2. nervous system cells

3. Y

4. N

adeno-associated virus

1. Max Gene Size and Nucleic Acid Type

2. Cell Specificity

3. trigger an immune response? (Y/N)

4. does it integrate into host cell’s genome? (Y/N)

1. 5 kb; DNA

2. can enter any cell type but engineer can choose; can engineer proteins on surface to target specific cells (gives the scientists/doctors more control)

3. N

4. Y

adenovirus

1. Max Gene Size and Nucleic Acid Type

2. Cell Specificity

3. trigger an immune response? (Y/N)

4. does it integrate into host cell’s genome? (Y/N)

1. 7500 kb; DNA

2. can enter any cell type but engineer can choose; can engineer proteins on surface to target specific cells (gives the scientists/doctors more control)

3. Y

4. N

retrovirus

1. Max Gene Size and Nucleic Acid Type

2. Cell Specificity

3. trigger an immune response? (Y/N)

4. does it integrate into host cell’s genome? (Y/N)

1. 8000 kb; RNA

2. can enter any cell type but engineer can choose; can engineer proteins on surface to target specific cells (gives the scientists/doctors more control)

3. Y

4. Y

lentivirus

1. Max Gene Size and Nucleic Acid Type

2. Cell Specificity

3. trigger an immune response? (Y/N)

4. does it integrate into host cell’s genome? (Y/N)

1. 8500 kb; RNA

2. can enter any cell type but engineer can choose; can engineer proteins on surface to target specific cells (gives the scientists/doctors more control)

3. N

4. Y

Pros and Cons for Plasmid

Pros:

• works with any gene

• gene size doesnt matter

• no danger to patient (no immune response)

Cons:

• cells may not uptake the free floating DNA

• plasmid can degrade quickly → treatment is temporary

• protein production (products) is low

Pros and Cons for Liposome

Pros:

• works with any gene

• gene size doesnt matter

• no danger to patient (no immune response)

Cons:

• cells may not uptake the free floating DNA

• Can degrade quickly → treatment is temporary

• protein production (products) is low

• lipsomes can sometimes be toxic to the patient

Pros and Cons for Herpes Virus

Pros:

• remain active for a very long time, even if not integrating into genome

• treatment has longer to work

• has a target cell type (nervous system cells)

Cons:

• cells may not uptake the free floating DNA

• Causes an immune response

• Temporary

  • Low protein production

Pros and Cons for Adeno-associated Virus

Pros:

• Engineer to target

• No immune response

• Long lasting treatment bc it integrates into genome

• Enters dividing and nondividing cells

Cons:

• Needs helper virus for replication

• helper may cause immune response ;w;

• small size

Pros and Cons for Adenovirus

Pros:

• Has a target cell type

• High protein production b/c it enters dividing and non dividing cells

Cons:

• No integration → temporary

• may cause immune response

Pros and Cons of Retrovirus

Pros:

• Has a target cell type

• Long lasting in genome (long lasting effects)

Cons:

• Enters only dividing cells (actively mitotic)

• Integrates into genome at random→ leads to other problems if it integrates at a place disrupting an important gene

• Low protein production (depends on where it integrates)

• may cause an immune response

• Needs reverse transcription (because it is RNA)

Pros and Cons of Lentivirus

Pros:

• Target specific cell

• Integrates→ long lasting

• Enters dividing and non dividing cells

• No immune response

Cons:

• random integration (leads to other problems if it integrates at a place disrupting an important gene)

• needs reverse transcription because it is RNA