The Role of PNA in Gene Therapy

Peptide Nucleic Acid (PNA)

Peptide Nucleic Acid (PNA) is a hybrid molecular technique which combines nucleic materials and proteins. The central dogma of life recognizes DNA as the genetic material within the cell. It is transcribed to produce messenger RNA which is then translated to proteins which play essential roles within the cell. mRNA expression is associated with the expression of specific genes which play critical roles within the body which is commonly reflected as a phenotypic characteristic. PNAs, a synthetic hybrid molecule has a peptide backbone directly connected to a nucleic acid strand. PNAs can bind to nucleic acid in a complementary manner as in the Watson-Crick base pairing making it one of the most suitable targets which could be exploited in either the transcription or translation process. If the PNA molecule directly binds to mRNA it immediately results in its degradation by a number of enzymes including ribonucleases. PNAs also acts to control transcription by directly blocking the action of RNA polymerase (Nielsen 67). PNAs can also utilize triplex invasion to promote transcription of specific genes. PNAs also interferes with the translation process ultimately affecting the expression of specific proteins (Nielsen 69). The mechanism suggested above can be utilized in the development of different drugs. For example, PNAs could be utilized to develop oligomer based drugs which affect the activity of RNA by directly binding onto the translated molecule and as a result causes the degradation of the molecule by a number of enzymes. In some instances, blocking the expression of the expression of a specific mRNA molecule results restores the functions of a cell especially if the molecule was specifically coding for specific products associated with a disease.

Work Cited

Nielsen, Peter E. “A new molecule of life?.” Scientific American 299.6 (2008): 64-71.