B6.3.18

Question 1

What is gene therapy

Answer 1

The placement of a fully functioning allele into a cell containing a faulty allele for the same gene. Gene therapy would involve placing the normal version of the gene.

Question 2

What are the main steps in gene therapy

Answer 2

Cutting out the normal version of the gene from the DNA of a healthy person using restriction enzymes

Producing many copies of the normal allele

Inserting copies of the normal allele into the cells of a person with the genetic disorder. This can be achieved by injecting a virus, which is modified to carry the allele, into the body. When the virus infects body cells, DNA is placed into them.

Question 3

What is the main difficulty in gene therapy

Answer 3

The healthy alleles may not go into every target cells
The healthy alleles may join chromosomes in random places so they may not work properly
Treatment may not only be short lived, as treated cells may be replaced naturally by the patients own untreated cells.

Question 4

What are future advances in medicine to be made

Answer 4

Locating genes that might be linked to inherited diseases. This allows doctors to identify people who are at higher risk of a disease before the disease occurs, allowing preventative steps to be taken.

Developing drugs that directly target disease-causing genes, or the proteins they code for

Developing new gene therapy treatments for diseases that currently have no cure

Developing personalised medicines. A knowledge of your genetic and molecular make-up allows doctors to prescribe medicines suited to an individual.