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Gene therapy

Gene therapy is an experimental technique that uses genes to treat or prevent disease. The scientific techniques developed in the 1980’s and 1990’s to allow investigation of the structure of DNA, first discovered by Crick and Watson in 1953. There have also been significant advances made in analysis of human genes (The Global Human Genome Programme). The use of these and other techniques have allowed the genes and gene disorders to be identified and tracked through families with great accuracy. 

The genes, some of which have been identified for the first time using the new techniques, In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including:
  • Replacing a mutated gene that causes disease with a healthy copy of the gene.
  • Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
  • Introducing a new gene into the body to help fight a disease.

GENES are made of DNA – the code of life. They are made of a sequence of chemicals
with the initials A, C, G and T, just like the alphabet makes words and sentences which can be turned into instructions. Everyone inherits genes from their parents and passes them on in turnto their children. Our cells are divided into two groups, the somatic cells which make up the working parts of the body, and the germ cells (or sex cells: sperm in men and eggs in women) which pass on genetic material to our children. Every normal somatic cell contains the same coded DNA instructions, even if only some of them are used.
              It will only change cells in the body of the person being treated, and not be passed on to children. Somatic gene therapy can be targeted to, for example, the liver, blood or lungs, to correct a medical problem which already exists and which can be treated by the gene or its protein product, such as an inherited disease or cancer.
Sperm and egg cells are different. They are the cells which go to form the individuals of
the next generation and pass the genes from both parents to the offspring. They are the GERM CELLS.
                   Would involve the deliberate insertion of a gene into the germ cells, deletion of a gene from them, or alteration of a gene already there. Most people agree that germ line therapy raises serious ethical issues, since changes would be inherited.

                   Pharmacogenomics is the study of how an individual’s genetic inheritance affects the body’s response to drugs. The term comes from the words pharmacology and genomics and is thus the intersection of pharmaceuticals and genetics. Pharmacogenomics combines traditional pharmaceutical sciences such as biochemistry with annotated knowledge of genes, proteins, and single nucleotide polymorphisms. 
  • More Powerful Medicines
  • Better, Safer Drugs the First Time
  • More Accurate Methods of Determining Appropriate Drug Dosages
  • Advanced Screening for Disease
  • Better Vaccines
  • Improvements in the Drug Discovery and Approval Process
  • Decrease in the Overall Cost of Health Care
  • Complexity of finding gene variations that affect drug response
  • Limited drug alternatives
  • Disincentives for drug companies to make multiple pharmacogenomic products.
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