Gene Therapy Introduction

What are genes?

• Genes are present inside the chromosome, which are the basic physical and functional units of hereditary.
• Genes are made up of specific sequences of bases and these gene bases, code for specific protein production as needed by the human body for normal metabolism.
• Proteins are required for normal cellular function, proper functioning of proteins is required for production and maintaining of cellular structures.
• When alterations in gene takes place the proteins which are coded by these gene are not able to function and causes genetic disorders.
• Gene therapy is done to repair the defective genes responsible for disease. Gene therapy is done by:

1. Inserting the normal gene into the genome to replace the non functional gene.
2. By recombination: The abnormal gene is replaced by normal gene.
3. By selective reverse mutation: The abnormal gene is repaired to become normal and perform its normal function.