Skip to main content

A “CRISPR” way to study disease

Jun. 11, 2015, 10:00 AM

by Henry H. Ong

Human diseases often result from complex genetic interactions. A common way to study these interactions is to simultaneously turn off multiple genes, preferably with temporal and spatial precision. However, current methods for this approach, called multiplex conditional mutagenesis, take a large amount of time and resources.

Wenbiao Chen, Ph.D., and colleagues have developed a faster and simpler method. Reporting last month in Genetics, they employed the CRISPR/Cas9 system – a revolutionary technology that greatly accelerates gene manipulation – to perform multiplex conditional mutagenesis in a common human disease model – the zebrafish.

By tailoring the two components of the system, cas9 and guide RNA, to be tissue-specific and inducible, they showed liver-specific mutagenesis of insulin receptor genes in just one generation resulting in impaired glucose homeostasis. They also showed retinal-specific mutagenesis of the ascl1a gene leading to abnormal photoreceptor regeneration in the eye.

This technique can potentially be a valuable tool for advancing our understanding of the genetic basis of human diseases.

The research was supported in part by grants from the National Institutes of Health (DK088686, EY024354) and from the American Diabetes Association.

Send suggestions for articles to highlight in Aliquots and any other feedback about the column to aliquots@vanderbilt.edu

Recent Stories from VUMC News and Communications Publications

Sharon Seibert is among the more than 5,000 patients who have received a stem cell transplant at Vanderbilt-Ingram Cancer Center, which has one of the best survival rates in the nation and is at the forefront of new cellular therapies.

Momentum

Sharon Seibert is among the more than 5,000 patients who have received a stem cell transplant at Vanderbilt-Ingram Cancer Center, which has one of the best survival rates in the nation and is at the forefront of new cellular therapies.

The first few minutes of Charlie’s life were a blur, as a team of doctors and nurses at VUMC worked to resuscitate him and stabilize his heart rate. He was then transferred to the Neonatal Intensive Care Unit at Monroe Carell Jr. Children’s Hospital at Vanderbilt.

Hope

The first few minutes of Charlie’s life were a blur, as a team of doctors and nurses at VUMC worked to resuscitate him and stabilize his heart rate. He was then transferred to the Neonatal Intensive Care Unit at Monroe Carell Jr. Children’s Hospital at Vanderbilt.

Tucked away in a Vanderbilt conference room, 36 adults huddle over Lego pieces. Eleven teams have been assigned to assemble multicolored Legos using the written directions included in the packet. The result should be a Frankenstein figure.

Vanderbilt Nurse

Tucked away in a Vanderbilt conference room, 36 adults huddle over Lego pieces. Eleven teams have been assigned to assemble multicolored Legos using the written directions included in the packet. The result should be a Frankenstein figure.

Marissa Benchea has CF, and she is one of hundreds of thousands of adults not only surviving but thriving with a chronic childhood disease.

Vanderbilt Medicine

Marissa Benchea has CF, and she is one of hundreds of thousands of adults not only surviving but thriving with a chronic childhood disease.

more