Gene Editing Breakthroughs Accelerate Heart, Stroke Research

Gene Therapy

Gene therapy research continues to deliver breakthroughs this week with the development of a CRISPR-edited droplet that could lead to a cure for heart disease and the possibility of turning glial brain cells into neurons to restore visual and motor functions. 

Scientists from the University of Utah have developed a new technology that enables the Nobel Prize-winning CRISPR system to combine droplet microfluidics, multiple ribonucleoprotein injections and DNA barcoding into microscopic oil-encased droplets that enable large-scale genetic screening in animals. 

Previously, the task was considered too laborious because each gene study had to have its own inactivation and research process, which made things extra difficult if 100 analyses were conducted. Keeping track of large numbers of mutant animals was immensely prohibitive given the amount of time, manpower, and resources it exhausted.

With this new development, the researchers created the Multiplexed Intermixed CRISPR Droplets, aptly called MIC-Drop, to bring gene target in one needle without affecting the other contents. To establish a screen of multiple genes, a library of zebrafish RNAs was created, each packaged into its own droplet with the Cas9 enzyme and a DNA barcode for identification. 

By injecting the droplet into hundreds of zebrafish embryos, the researchers were able to distinguish which of the animal's genes might contribute to heart development and, hopefully, treatment. Zebrafish genes are a lot similar to human genes, so being able to move on to a mass study is a milestone for heart disease therapy. 

Meanwhile, another group of scientists from Purdue University and Jinan University has discovered a way to use gene editing to transform glial brain cells into neurons with the hope of restoring visual and motor function in patients who had suffered a stroke. Because neurons by themselves cannot regenerate, reprogramming local glial cells and turning them into functioning neurons could be the key to preventing physical disablement. 

The researchers are evaluating outcomes on mouse models as of this writing, but if the study continues to provide positive results, it might not be long before human trials are started. Current interventions involve stem cell therapy but finding an immune match for the treatment to succeed is often cumbersome and difficult. 

"We are directly reprogramming the local glial cells into neurons. We don't have to implant new cells, so there's no immunogenic rejection. The process is easier to do than stem cell therapy, and there's less damage to the brain," said Alexander Chubykin, the associate professor of biological sciences who leads the University of Purdue team. 

"We are helping the brain heal itself. We can see the connections between the old neurons and the newly reprogrammed neurons get reestablished. We can watch the mice get their vision back," he continued. 

Visual function is easier to measure accurately compared to motor skills, but both are common to people who have had a stroke. The scientists are looking into the effectiveness of this procedure in live mice using advanced optical imaging tools. 

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