Inquiring Minds


Photo by Daniel Dubois.

RNA Interference Heals Growth Deficiency Disorder

Vanderbilt researchers have demonstrated for the first time that a new type of gene therapy called “RNA interference” can heal a genetic disorder in a live animal.

Their study, published last fall by the journal Endocrinology, shows that RNA interference can “rescue” a strain of mouse that has been genetically engineered to express a defective human hormone that interferes with normal growth. When the gene that produces the defective human growth hormone is inserted into the mouse’s genome, it also stunts the mouse’s growth. But when a small snippet of RNA that interferes with the hormone’s production is also added, the mouse is restored to normal.

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Liver Allocation System Lowers Death Rates

Vanderbilt researchers have found that the United Network for Organ Sharing’s (UNOS) adoption of an objective-only method of allocating donated livers has lowered the number of deaths among patients on the waiting list. In 2002, UNOS adopted a system using laboratory-based values to characterize a patient’s need for liver transplantation.

Previously, patients who spent the longest time on the waiting list for a liver were often given priority. After the change, wait times became less of an issue while severity of condition was prioritized.

The change was the subject of great debate and prompted Vanderbilt researchers to examine the outcomes associated with the new liver allocation policy. Results of the study were released last fall in the Archives of Surgery.

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Melatonin Study Could Help Children with Autism

Vanderbilt sleep researchers are reporting a relationship between good sleep and how much melatonin the body produces–the first in a series of research studies intended to help children with autism spectrum disorders (ASD) sleep through the night.

“This suggests that children with ASD who have decreased melatonin levels have decreased levels of deep sleep,” says lead author Dr. Beth Malow, director of the Vanderbilt Sleep Disorders Center. “We didn’t actually give the supplement; we measured natural levels of melatonin in the body. One could infer, based on what we found, that a supplement might be good.”

More research is needed before recommending that children begin taking melatonin supplements to benefit their sleep.

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