Gene therapy is the insertion of a normal allele of a gene into cells to replace a faulty allele that causes an inherited disorder. This could be done in the very early embryo (germ line therapy) or in the affected body parts (somatic therapy)
Somatic Therapy
- Step 1- identify the gene involved, for example the gene for CF is found on chromosome 7.
- Step 2- make copies of the normal allele and insert into a vector- the two most common vectors for human cells are viruses and lipsomes.
- Step 3- use the vector to insert the allele into the target cells.
Cystic Fibrosis
Trials of gene therapy for CF have successfully transferred the normal CFTR allele to the epithelial cells in the lungs of CF patients. After insertion of the normal allele into the genome of the target cell it can make the functioning CFTR and therefore allow normal chloride movement.
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