Novel gene therapy on the horizon, says Brenner Children's Hospital pediatrician

in cells and affect 1 in every 12,000 people in the United States each year.

Payne and his colleagues have spent the last 36 months perfecting the delivery of the proteins across cell membranes and are ready to begin animal studies on the technology once they receive additional research funding.

"This technology could replace the current gene therapy being used in a much safer way," he said. "And while this study is aimed at preventing deaths in infants, potentially many adults could benefit from this. Over the span of a lifetime, many adults will accumulate defects in the mitochondria, which show up as heart and brain diseases later."

Brenner Children's Hospital is part of Wake Forest University Baptist Medical Center. Colleagues who collaborated with this project include Victoria Del Gaizo, Payne's graduate student, and Jamal Ibdah, M.D., associate professor of gastroenterology at Wake Forest.


Contact: Rae Beasley
Wake Forest University Baptist Medical Center

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