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Emily Nurre, MD/MPH Candidate

Award Name TL1

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Adeno-associated virus serotype 9 for Spinal Muscular Atrophy (SMA)

The Ohio State University’s Center for Clinical and Transitional Science has awarded Emily Nurre, MD/MPH Candidate, with a TL1 Award for her research involving ‘Safety assessment of intravascular administration of scAAV9-SMN.’

In her research, Nurre uses a mouse model to determine whether gene therapy, utilizing adeno-associated virus serotype 9 (AAV9), is safe to use in potential clinical trials for spinal muscular atrophy (SMA). Nurre performs hematological and histopathologcial studies on a cohort of mice. In these studies, Nurre checks the complete blood count, liver function tests, creatine kinase levels and histopathology. Additionally, she completes behavioral studies and weighs all animal models weekly.

Not only is Nurre doing her research in mice, but she also studies primates for a closer approximation of how the gene therapy will behave in humans.

“Research in mice is important for toxicology data,” Nurre said. “From the primate studies, we are able gather further information regarding safety and transduction pattern in each species.”

The goal of Nurre’s research is to focus on a potential gene therapy for neuromuscular disorders and determine if AAV9 combined with SMN (the gene depleted in SMA) is safe enough to progress toward clinical trials for patients with Type 1 SMA.

Nurre became interested in this subject through her passion for neuroscience. She has presented her research at the National meeting for the American Academy of Neurology and has completed her MPH in clinical translational science.

Data collected thus far shows no adverse effects due to therapy. “My hope is that my research will help advance the field of gene therapy,” Nurre said. “Specifically, I hope that this type of gene therapy shows potential for future treatment for SMA.”

By Charaun Little, Monday, February 6, 2012

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