Genetic engineering is applied to produce a recombinant human protein to treat people with ADA-SCID. Outline the principles of genetic engineering.
Explain why a single infusion of gene-corrected stem cells is enough to cure the disease.
Explain why a promoter must be transferred in addition to the target gene.
A modified retrovirus is employed to place the new gene into the DNA of the blood stem cells. State two ethical considerations of using a retrovirus for gene therapy.
The gene therapy technique used to cure ADA-SCID is not suitable for treating the genetic disease called Huntington’s disease. A newer technique called gene editing could potentially be used instead to cure Huntington’s disease. Explain why gene editing is more suitable as a potential cure for Huntington’s disease.