The dangers and concerns of gene therapy.

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The dangers and concerns of gene therapy

Many diseases are genetically determined; for example, cystic fibrosis (CF). Others are influenced by genes, but involve additional factors as well; for example, cancer and diabetes mellitus. Recent scientific developments make it possible to target such genetically related diseases using gene therapy. Gene therapy is transferring the normal allele into to the cells to produce the correct proteins that the mutated allele does not.  Under UK legislation altering genes of somatic cells (body cells) to treat a disease in an individual is permitted.  This is done mainly in two ways.  For example, treating cystic fibrosis can be done using liposomes, the normal cystic fibrosis transmembrane regulatory protein allele can be transferred into lung epithelial cells as follows.

  1. A normal allele is inserted into a loop of DNA which is called a plasmid
  2. The plasmids are combined with liposomes (spherical phospholipid bilayers) to form a liposome - DNA complex
  3. The complexes are breathed into the lugs using a nebuliser.  Inside the lungs the liposomes fuse with the cell membrane
  4. The DNA is can then be carried into the cell
  5. The normal allele is transferred to the nucleus where it is transcribed
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Functional cystic fibrosis transmembrane regulatory channel proteins are produced and incorporated into the cell membranes,alowing chloride ions to leave the cell thus preventing the thick sticky mucus asociated with the symptoms of cystic fibrosis.  The treatment is only temporary because the epithelial cells in the airways die and the new cells in their place do not contain the normal allele.  The longest correction in the trials has lasted for about two week 2 weeks, so patients would have to be treated regularly throughout their lives.

An alternative method uses a virus called adeno-associated virus (AAV). The AAV does ...

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