Targeted Inactivation of Genes in Animal Model

Researchers at the University of Massachusetts Medical School (UMMS) report on a new technique that improves the ability of scientists to target individual genes for inactivation—a technique with broad implications for both basic science research and human disease. Two scientific teams at UMMS -- one led by Scot A. Wolfe, PhD, and the other by Nathan D. Lawson, PhD -- working with the zebrafish developed a method to create and deliver a tailor-made “restriction enzyme” that inactivates a specific gene in a zebrafish embryo.

Their research article appears as an advance online publication of the journal Nature Biotechnology.

“The best way to figure out what a gene does in an organism is to replace it with a non-functional version, breed the individual, and then look at the offspring to see what’s wrong with them,” said Laurie Tompkins, PhD, who oversees genetic mechanisms grants at the National Institute of General Medical Sciences, “The problem is that it’s hard to swap in non-functional genes that are inherited by the offspring. These investigators have devised a way to do this, which will enable many scientists to answer questions that were previously out of reach.”

“We believe that this work will fundamentally change how researchers make knockouts—research organisms in which one or more genes have been genetically engineered to be turned off—in many model organisms,” said Dr. Wolfe. “In this paper, we demonstrate the feasibility of this approach for gene inactivation using the zebrafish, but we believe that this technology should be applicable to other vertebrate and non-vertebrate systems with exciting implications for the development of new models for the study of human disease.”

This article was adapted from a press release issued by University of Massachusetts Medical School