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Novel Gene Therapy on the Horizon, Says Brenner Children’s Hospital Pediatrician

WINSTON-SALEM, N.C. – Researchers at Brenner Children’s Hospital are working on a novel gene therapy to help new moms and their babies who suffer from genetic defects, according to findings reported at the Society of Pediatric Research in Seattle today.

R. Mark Payne, M.D., a pediatric cardiologist and researcher at Brenner Children’s Hospital, has discovered a new way to transmit proteins across cell membranes – a finding that in the future might prevent or treat diseases like Sudden Infant Death Syndrome (SIDS) and Trifunctional Protein (TFP) deficiency in infants and their mothers.

“The treatment involves getting the right protein into the cells or mitochondria they need to function properly,” Payne said. “Mitochondria produce most of the energy within cells and defects in their function are among the most common genetic defects in the United States and currently can not be prevented.”

Some genetic defects occur when mitochondria lack a certain protein, which cause babies, and in some cases their mothers, to become sick and die, Payne said. Current gene therapy attempts have tried injecting the patient with a virus that corrects the defect. However, the effectiveness of this approach is very poor and the side effects aren’t good – patients may die from the treatments, according to Payne.

“Instead of injecting a virus into the cells, we want to take the needed protein and inject only that protein into the cells,” Payne said. “This is highly efficient technology – the needed proteins get absorbed and are positioned correctly within the mitochondria located in the cell, which is necessary to correct the defect.”

Payne and his colleagues say this new technology could mean that mothers suffering from genetic abnormalities such as TFP may be able to receive injections until their due date – preventing premature delivery and sometimes death for both the mother and infant.

TFP is one of many genetic defects that involve mitochondria 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.

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Media Contact: Rae Beasley (336) 716-6878, rabeasle@wfubmc.edu; Karen Richardson (336) 716-4453, krchrdsn@wfubmc.edu; or Barbara Hahn (336) 716-6877, bhahn@wfubmc.edu.

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