Synthetic biology yields new approach to gene therapy

Bioengineers at The University of Texas at Dallas have created a novel gene-delivery system that shuttles a gene into a cell, but only for a temporary stay, providing a potential new gene-therapy strategy for treating disease. The approach offers distinct advantages over other types of gene therapies under investigation, said Richard Taplin Moore MS’11, a doctoral student in bioengineering in the Erik Jonsson School of Engineering and Computer Science. He is lead author of a study describing the new technique in the Jan. 30 issue of the journal Nucleic Acids Research.

“In other gene therapy approaches, the therapeutic genetic messages being delivered can persist for a long time in the patient, potentially lasting for the patient’s entire lifetime,” Moore said. “This irreversibility is one reason gene therapies are so difficult to get approved.”

The UT Dallas study describes proof-of-concept experiments in which a gene carrying instructions for making a particular protein is ordered to self-destruct once the cell has “read” the instructions and made a certain quantity of the protein. In its experiments with isolated human kidney cells, the research team successfully delivered—and then destroyed—a test gene that makes a red fluorescent protein. Via Synthetic biology yields new approach to gene therapy.

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