Gene Therapy

Gene therapy

functioning allele is inserted into cells that have faulty/ non-functioning copies of an allele

positives of gene therapy

-potential to cure uncurable diseases

negatives of gene therapies

-effect is short-lived

-immune response to allele or vector can occur

-the recombinant allele may not be expressed

only type of genetic disorders gene therapy can be used on

genetic disorders caused by recessive alleles of a single gene

How cystic fibrosis could be cured

replacing the CFTR gene by introducing a non-faulty gene into the lungs

liposome

balls of lipid containing genes allowing easy transport across cell surface membranes

how gene therapy could be introduced into a cystic fibrosis sufferers body to get best and quickest effect

nasal spray straight into respiratory system