Name the target gene and restriction step, form the recombinant vector, and explain how transformation or viral delivery places DNA into the host cell for expression or therapy.
Alpha-1 antitrypsin (AAT) deficiency is an inherited disease that can affect different organ systems.
Alpha-1 antitrypsin (AAT) is a protein produced in the liver.
One potential treatment for AAT deficiency uses adenoviruses to genetically modify cells in an affected person.
Adenoviruses contain double-stranded DNA.
Explain how adenoviruses can be used to treat AAT deficiency in a person.
An answer that includes four of the following points:
- \{identification / isolation \} of (corrected) AAT gene (1)
- description of one enzyme used to insert gene (1)
- adenoviruses can enter host cell (1)
- and combine their own genetic material with that of the cell (1)
- therefore a functional AAT protein can be produced (1)
E.g. cut the adenovirus DNA using the same restriction endonuclease / insert the gene
into the adenovirus DNA using
DNA ligase adenovirus acts as a vector
(4)