Gene Therapy - The Future Is Here! (cont.)

Uses of gene therapy

Gene therapy is being used in many ways. For example, to:

  • Replace missing or defective genes;
  • Deliver genes that speed the destruction of cancer cells;
  • Supply genes that cause cancer cells to revert back to normal cells;
  • Deliver bacterial or viral genes as a form of vaccination;
  • Provide genes that promote or impede the growth of new tissue; and;
  • Deliver genes that stimulate the healing of damaged tissue.

A large variety of genes are now being tested for use in gene therapy. Examples include: a gene for the treatment of cystic fibrosis (a gene called CFTR that regulates chloride); genes for factors VIII and IX, deficiency of which is responsible for classic hemophilia (hemophilia A) and another form of hemophilia (hemophilia B), respectively; genes called E1A and P53 that cause cancer cells to undergo cell death or revert to normal; AC6 gene which increases the ability of the heart to contract and may help in heart failure; and VEGF, a gene that induces the growth of new blood vessels (angiogenesis) of use in blood vessel disease.

A short synthetic piece of DNA (called an oligonucleotide) is being used by researchers to "pre-treat" veins used as grafts for heart bypass surgery. The piece of DNA seems to switch off certain genes in the grafted veins to prevent their cells from dividing and thereby prevent atherosclerosis.

Delivery of genes into cells

Gene delivery can be used in cells that have been removed from the body (ex vivo gene therapy) or in cells that are still in the body (in vivo gene therapy). Genes can be delivered into cells in different ways. The selection of a gene delivery system depends on the target cell, the duration of gene expression required for therapeutic effect, and the size of the piece of DNA to be used in the gene therapy.

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