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| Edited By: |Source: ANI |Updated: Mar 11, 2018, 02:42 AM IST
Scientists at Ohio State University say that a lethal childhood disorder spinal muscular atrophy may be susceptible to effective treatment through gene therapy.
Research in mice showed that the therapy could reverse a protein deficiency characteristic of the condition and thus correct motor function, restore nerve signals and improve survival.
The mice were engineered to develop the disorder. The disease results when due to a missing gene, motor neurons - nerve cells that send signals from the spinal cord to muscles - produce too little of what is called survival motor neuron protein.
The researchers used an altered virus to deliver a portion of DNA that makes the protein into the veins of 1- to 10-day-old mice.
The virus injected into the youngest mice reached almost half of their motor neurons, resulting in improved muscle coordination, properly working electrical signals to the muscles and longer survival, scientists said.
"When you put the protein in postnatally, it will rescue the genetic defect. This technique corrects the mice considerably more than any drug cocktails being studied as a potential treatment in humans," said Arthur Burghes, biochemist at Ohio State University and a senior co-author of the study
Gene therapy is a relatively new class of experimental treatments involving introducing genes into patients through various means in order to replace defective genes or otherwise help fight illness.
Although gene therapy has sparked great excitement among scientists, the research has suffered setbacks, including a 1999 death during a clinical trial for ornithine transcarboxylase deficiency.
Other studies have shown better results, but the U.S. Food and Drug Administration has to date not approved any gene therapy treatments.
The scientists used a special form of a virus to deliver the protein to nerve cells in the mice.
Brian Kaspar, an investigator in the Research Institute at Nationwide Children's Hospital and assistant professor of pediatrics at Ohio State, also a senior co-author of the study, said that this virus still has the capability to infect cells but has been altered so it will not copy itself and cause illness in humans.
The study has been published online in the journal Nature Biotechnology.
