Muscle diseases are a serious health problem that affect millions of people worldwide. To understand how these diseases develop and how to treat them, scientists need to study the structure and function of skeletal muscle, the type of muscle that enables movement.

However, studying muscle is not easy, as it requires the use of mice as model organisms and a lot of time and resources.

But now, researchers at the University of Basel have developed a new method that could revolutionize muscle research.

This method is not only faster and more efficient than conventional ones, but also greatly reduces the number of experimental animals needed for studying the function of genes in muscle fibers.

The results of their study have been published in Nature Communications.

New method combines CRISPR/Cas9 and AAV to alter gene function in muscle fibers
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The new method is based on the combination of two existing techniques: the CRISPR/Cas9 method and the adeno-associated virus (AAV) method.

The CRISPR/Cas9 method is a powerful tool that allows scientists to alter gene function in cells by using a protein called Cas9 and a guide RNA that recognizes a specific DNA sequence.

The Cas9 protein cuts the DNA at the target site, causing changes in gene expression or function.

The AAV method is a way of delivering genes or other molecules into cells by using a harmless virus that infects muscle cells.

The researchers at the University of Basel used these two methods together to study gene function in muscle fibers.

First, they bred mice that had the Cas9 protein already present in their muscle fibers, but not in other organs. Then, they injected the mice with an AAV that carried the guide RNA for the gene they wanted to study.

This way, the guide RNA met the Cas9 protein only in the muscle fibers, where they changed the gene function as desired.

The benefits of the new method for muscle research

The new method has several advantages over conventional methods for muscle research.

Faster and more efficient

The researchers were able to change gene function in muscle fibers within two weeks after injecting the AAV, whereas conventional methods would take months or years to achieve the same result.

More precise and specific

The new method allows scientists to target only muscle fibers and not other organs or tissues, avoiding unwanted side effects or interference from other genes.

Ethical and economical

The new method reduces the number of experimental animals needed for studying gene function in muscle fibers by up to 90%. This means less animal suffering and less cost for animal care and maintenance.

The new method also opens new possibilities for studying multiple genes or signaling pathways simultaneously or sequentially in muscle fibers, which could lead to new insights into muscle biology and disease.

Potential applications of the new method for muscle biology and disease

The new method could have a significant impact on muscle research and beyond. It could help scientists to better understand how muscle diseases develop and progress, how aging affects muscle function and regeneration, and how exercise or drugs influence muscle performance and health.

The new method could also be applied to other tissues or organs that are difficult to study with conventional methods, such as the brain, heart, or liver.

The new method is a win-win situation for both science and society: it enables faster and more efficient research with fewer laboratory animals, while advancing our knowledge and improving our health.