Targeting mRNA to Stop Genetic Diseases

Friday, June 17, 2011

Correcting Genetic Flaw

Scientists at the Rochester University have come up with a cure for genetic faults which leads to diseases, by changing the genetic code. This can help researchers reverse the course of genetic disorder like cystic fibrosis, muscular dystrophy and cancer which occurs due to faults in the genetic code of a person.

The genetic code of a body is its instruction manual that instructs a cell on how to make a specific protein. The growth and development of a body depends on how these cells read the genetic instructions from DNA. The messenger RNA (mRNA) functions as an intermediary in this process. Researchers involved in the project have artificially modified RNA and in doing so changed the original instructions from the DNA to create protein. This resulted in the creation of a new protein which can be used to treat debilitating and fatal genetic diseases among individuals.

The production of proteins however, can be affected by faults or mutations in the code which can lead to a flawed protein. In this study, scientists focused on a common type of mutation that occurs when an mRNA contains a pre-mature stop signal. This premature stop signal orders a cell to stop reading the genetic information which hampers the creation of protein.  However, scientists were able to reverse this signal into a ‘go’ signal, thus allowing the cell to read the genetic information that can aid in the creation of protein.

This is a remarkable achievement made by modern science as it has the potential to treat one third of genetic diseases which are caused by pre-mature stop signals. The team also used another type of RNA- the guide RNA to modify mRNA which allows them to specifically focus on one genome and make the desired changes. They claimed to have performed this experiment both in test tube and in live yeast cells.  

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