Genetic therapies

Question 1

What is gene therapy?

Answer 1

The transfer of DNA or RNA into cells of an organism to treat disease or to mark cell populations

Question 2

What is the more preferred gene therapy?

Answer 2

Gene augmentation over gene replacement

Question 3

What diseases might gene therapy be used for and what is the criteria?

Answer 3

Immunodeficiences, CF, some cancers

Criteria is that it must be a single gene disorder; life threatening;

Question 4

How can we get genes into cells?

Answer 4

Physically- electroporation

Vectors- VIRUSES eg retrovirus, herpes 1 virus

Question 5

How could mutation correction in vivo be attained?

Answer 5

Target cells at DNA levle, uses homologous recombination; RNA level targeting ribozymes/RNA. Editing

Question 6

What is targeted inhibition?

Answer 6

Inhibition of gene at DNA level, RNA level and protein level. Good for gain of function mutations