Genes and gene expression

Gene therapy is the process whereby a therapeutic protein is synthesized fr om a DNA molecule (gene) that has been inserted into the cells. The goal is to produce the desired protein in the proper quantity in the proper locati on, Successful designing of vectors for gene therapy requires knowledge of gene structure and regulation. The gene is comprised of protein-coding sequ ences (called exons) that are interrupted by noncoding sequences (called in trons), The expression of a gene in a tissue is regulated by proteins that bind to specific deoxyribonucleic acid sequences generally upstream from th e first exon, in the deoxyribonucleic acid promoter domain. Understanding t he mechanisms of gene regulation provides the deoxyribonucleic acid sequenc es necessary to direct expression of specific genes in the right tissue at the proper time in the desired amount.