Miracle cures on the horizon for genetic disease

In a latest break through, scientists have found a new technique to alter the genetic code behind hundreds of diseases and this was hailed by U.S. scientists as a “miracle of modern medicine.” Genes responsible for devastating diseases can be corrected by controlling faulty messenger cells that are central to the body's protein production, researchers said Wednesday.

Dr. Robert Bambara, from the University of Rochester Medical Center said, “The ability to manipulate the production of a protein from a particular gene is the new miracle of modern medicine. This is a really powerful concept that can be used to try to suppress the tendency of individuals to get certain debilitating, and sometimes fatal, genetic diseases that will forever change their lives.”

For the study the researchers focused on genetic material called messenger RNA (mRNA) that can affect the production of vital proteins when a premature "stop" or "codon" signal occurs. This causes a cell to stop reading the genetic instructions partway through the process, resulting in the creation of an incomplete, shortened protein. The team then produced normal, healthy proteins in test tubes and in live yeast cells by using short strands of another type of mRNA to correct the faulty ones. The researchers were hoping to apply the same principles to the human body, with the aim of altering the course of genetic disorders such as cystic fibrosis, muscular dystrophy and some cancers. More than 8,000 people in Britain suffer from cystic fibrosis, including Gordon Brown's son, Fraser. A further 30,000 people have muscular dystrophy. Both diseases are highly debilitating and lead to shortened lifespans. The genetic code is the body's own instruction manual and tells it how to develop and how to function.

The study appeared in the journal Nature.

“This is a very exciting finding,” lead study author Professor Yi-Tao Yu said. “No one ever imagined that you could alter a stop codon the way we have and allow translation to continue uninterrupted like it was never there in the first place.”

Much more research is needed and, even if the method could be applied in humans, developing it into a safe, proven application for the treatment of human genetic diseases will take some time say researchers. At present there are no cures for genetic disease and the medical profession has concentrated on prevention, screening children in the womb for any risks.

Dr. Ananya Mandal

Written by

Dr. Ananya Mandal

Dr. Ananya Mandal is a doctor by profession, lecturer by vocation and a medical writer by passion. She specialized in Clinical Pharmacology after her bachelor's (MBBS). For her, health communication is not just writing complicated reviews for professionals but making medical knowledge understandable and available to the general public as well.


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