Gene Therapy

Question 1

Study showing the efficiency of gene therapy was named?

Answer 1

“Secretion of functional α1-antitrypsin is cell type dependent”

Question 2

Common Target for gene therapies is?

Answer 2

Muscle cells due to more easy access , but it is not much efficient

Question 3

What is AAT?

Answer 3

Alpha-1 Antitrypsin
a condition in which liver cells are unable to make adequate amounts of the protein AAT.

Patients are usually treated by receiving AAT via infusion

Question 4

Lack of AAT causes?

Answer 4

Chronic Obstructive Pulmonary Disease (COPD) or emphysema

Question 5

What is SAHA and what is the use?

Answer 5

suberoylanilide hydroxamic acid, or SAHA, helps muscle cells make AAT at a production level more like that of liver cells.

Question 6

the process that regulates proteins within the cell in order to maintain the health of both the cellular proteome and the organism itself. is called?

Answer 6

Proteostasis