Structural Biochemistry/Gene Therapy

The results of researches in Structural biochemistry have made the subject to be greatly linked with Pharmacy, Pharmacology, and Medicine. This is due to its ability to determine the three dimensional structures of proteins by using X-ray Chromatography and other techniques. These three dimensional structures say a lot about the functions of the proteins. Based on these structures, scientists are now able to find drugs and cures for different diseases. Gene therapy helps to correct mutated genes in human cells.

Genes are the building blocks of life, which are passed from the parent to the child. Genes are strung together to thread like structures called chromosomes. These human chromosomes are paired and hold innumerable genes. The genes and chromosomes are made up of tiny structures called deoxyribonucleic acid or DNA. The DNA is the blueprint of genetic information of previous generations. The genes determine the traits of an individual like physical features, habits, etc. There are approximately 25,000 – 35,000 genes in the human body. Unfortunately, sometimes the genes also pass on certain hereditary disorders from the parent to the offspring, which can be difficult to treat. This is when gene therapy comes in to play.

Gene therapy is a procedure where genetically mutated genes, which are the causative factors for numerous hereditary diseases, are corrected by injecting healthy genes into the body. The concept of treating these incurable diseases with the help of gene therapy was first proposed in the 1960’s. A group of scientists in the early 1970’s put forth a proposal where inherited disorders could be treated with the help of genes. The main aim of this therapy was to remove defective genes and replace them with healthy genes. In the year 1983, a few scientists from Baylor College of Medicine, Houston stated that a rare neurological disorder like Lesch Nyhan disease could be successfully treated with the help of gene therapy. A few years later, in 1990, an American doctor, W. Anderson conducted a study of gene therapy on a four year old girl who was suffering from a genetic disorder of the immune system.

With the help of gene therapy and embryonic stem cell research, many deadly and debilitating genetic disorders can be treated. The faulty genes undergo correction so that the coming generations are not carriers of mutated genes. Gene therapy has also come as a ray of hope for people suffering from Alzheimer's disease, Huntington's disease, Parkinson's disease and other such incurable genetic disorders.