lecture 21

Question 1

problem and solution of gene therapy being used to treat type one diabeties

Answer 1

the beta cells of the pancreas are destroyed, so no insulin is produced in the pancreas.
solution us to use gene therapy to place insulin producing genes into the liver.

Question 2

What is the general concept of gene therapy?

Answer 2

Use patients as the host for
protein production, so that we don’t have to administer repeated recombinant genes into people

Question 3

how would we use gene therapy for helping those with type one diabeties

Answer 3

So we instead put viral vectors into the liver as those cells still function. This means we place a liver promoter onto the plasmid so the gene is well expressed in the cell. For insulin treatment in the liver wed use glucose responsive promotor, so that the insulin is produced in response to high glucose.

Question 4

what does ex vivo mean

Answer 4

Ex vevo gene therapy means taking cells out of the body, like those of the bone marrow, and planting them with a viral plasmid. Then putting the cells with the new gene back into the person.

Question 5

what does in vivo mean

Answer 5

En vivo is directly putting the gene into the patient themselves.

Question 6

hwo does crispr work

Answer 6

Cas9 is a bacterial protein that functions to cut viral DNA in the cell. Cas9 can be programmed with a specific sequence of RNA. The RNA is complementary to a desired DNA sequence. Once the complementary DNA is found, it is cut by the protein. The cell then rebuilds the DNA. We can implant crispr with the desired fix to the DNA. Potentially repairing mutated genes with the correct nucleotides and changing a mutated gene product in the cell, curing a condition.