Bio 100 — Eat your heart out

134

by Dan Cole

BYU professor Steven Johnson and his staff are conducting research that has impact on gene therapy to cure debilitating genetic disease.

Gene therapy is an established field of medicine but poses difficult technical challenges for scientists today. The growing field only yields limited success yet researchers at BYU are laying the ground work for what could be the key to lasting success for patients. The BYU study may halt diseases such as cystic fibrosis, muscular dystrophy, hemophilia and other genetic diseases.

The major obstacle in gene therapy is initial treatment fails after only a few weeks. After the new genes are introduced into a host, the DNA becomes bundled around tight packs called chromatin. These bundled packs of chromatine known as nucleosomes become so tight that they inhibit the function of the implanted gene.

A new paper published in Nature magazine by Dr. Johnson and his colleges address how these nucleosomes are bundled and gives the scientific community a clue on a genome wide scale on how researchers could prevent things from being bundled.

“It’s not really understood why the DNA gets bundled up and turned off and what actually cause it or precipitates it to happen.” Johnson said. “What makes nucleosomes sit in a specific spot what anchors it in it’s particular place in the genome is the crux of how it will benefit potential gene therapy. If we can figure out what makes them sit in specific spots or what keeps them from sitting in specific spots, then we can use that for genes that are being put in to replace a defective gene.”

The paper is laying the ground work in defining what makes the architecture of the DNA in specific patterns. Geneticists will be able to manipulate or potentially leave areas open in that architecture that need to be activate or closed to eradicate defective genes.

Beth Cole PhD and Dean of the College of Nursing at BYU points out potential therapy may be able to be isolated to specific genetic groups.

“Medications that work for one group of patients, may not work for another group,” Cole said. “Some of these differences are genetically based. While we can now identify which genes are implicated in a particular illness or developmental problem, the focus of the next level of research is on how to alter the genetic structure to improve outcomes, or what other interventions modify an individual’s symptoms, long term adjustments, etc.”

 

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