AS Edexcel Biology unit 1 - Gene therapy

  • Created by: Hannah
  • Created on: 29-12-11 16:07

What is Gene therapy?

Gene therapy is the insertion of a normal allele into a target cell to replace a faulty allele that causes inherited disorders, such as cystic fibrosis.

This text refers to the use of gene therapy to assist the cure of CYSTIC FIBROSIS:

Somatic gene therapy

The normal allele is inserted into the target cell, the body part that is affected. This can be achieved either by inserting a genetically modified virus or by using a liposome (spherical phospholipid bilayer).


  • A copy of the normal allele is inserted into a plasmid (small…




I just wanted to clarify for anyone reading this that there is no cure for Cystic Fibrosis however scientists hope that gene therapy will one day improve enough to create a cure for CF.

Gene therapy has only really worked with cases of immunodeficiency but it has to be repeated due to inefficiency. It was tried with people with CF but was only 25% effective at creating the correct CFTR protein.

Good notes!






what does "the injected virus remains independant within the nucleus of the cells" actually mean?