Gene Therapy

Question 1

What is somatic cell gene therapy?

Answer 1

• it is the addition of a functioning copy of a faulty gene into the relevant specialised cells (augmentation)
• this means that lost polypeptides can now be synthesised and the cells can function normally

Question 2

How do you get the gene into the cell?

Answer 2

• by using a liposome (manufactured vesicle)

* liposome fuses with cell surface membrane releasing the gene inside the target cell

Question 3

What is germline cell gene therapy?

Answer 3

• involves germline cells (sperm, egg, zygote, stem cell)
• engineering a gene into any of these cells means that as an organism grows every cell contains a copy of the engineered gene which can function in any cell
• this can therefore be inherited by offspring
• currently illegal in humans

Question 4

What is gene therapy?

Answer 4

• it is treating genetic disorders by inserting new genes
• if the genetic disorder to caused by dominant alleles you can silence the dominant allele
• if the genetic disorder is caused by two recessive alleles you can add a working dominant allele to make up for them