CRISPR-Cas 9 Tool for Strengthening Muscles is a mRNA component that is mRNA-transferable

CRISPR-Cas 9 Tool for Strengthening Muscles is a mRNA component that is mRNA-transferable ...

Mutations that lead to muscle atrophy can be repaired with the gene editor CRISPR-Cas9. A team led by Helena Escobar has now introduced the tool into human muscle stem cells for the first time using mRNA, thus discovering a therapeutic therapeutic solution.

Despite this type of genetic defect, muscular stiffness and changes in function are expected to result in very similar results. Professor Simone Spuler, the head of the Berlin-based Max Delbruck Center for Molecular Medicine (MDC) and Charite Universitatsmedizin Berlin, believes the condition may be fatal especially if the respiratory or cardiac muscles are affected.

Chemothologists have already found this technique.

The majority of muscular dystrophies are incurable, and the result of Spuler''s recent paper, which is published in the journal Molecular Therapy Nucleic Acids, has opened the door to a clinical trial in which a therapy developed at the ECRC will be tested for the first time on patients with hereditary muscle atrophy. Dr.Helena Escobar, a postdoctoral researcher, has developed the idea of taking muscle stem cells from severed patients, using CRISPR

Escobar claims that when people returned to the laboratory, the scientists found that the technique applied in mice with muscle atrophy. Yet, the scientists discovered that the genetic guidelines for the gene editor were implemented in stem cells, using plasmids, which are circular, double-stranded DNA molecules. However, these plasmids could unintentionally integrate into the genome of human cells, which is also double stranded, and lead to undesirable consequences that are difficult to assess. This technique was ineffective in treating patients

Targeted correction of genetic defects

So the team decided to pursue a viable alternative: messenger RNA (mRNA), a single-stranded RNA molecule that recently received widespread approval as a major component of two Covid-19 vaccines. In the vaccines, the mRNA molecules contain the genetic guidelines for building the viruses spike protein, according to Christian Stadelmann, a doctoral student from the same university. In our experiments, we use mRNA molecules that contain the building instructions for the gene-editing tool.

The researchers employed a technique called electroporation, which temporarily transforms cell membranes into larger molecules. With the help of mRNA, the researchers demonstrated that the mRNA molecules entered almost all stem cells. So, the researchers investigated how the method may be used in a specific way.

A clinical trial is in the works

A technique similar to the CRISPR-Cas9 gene editor is capable of not cutting the DNA at one time with pinpoint accuracy. This allows us to experiment with even greater precision, yet this technique isn''t suitable for every muscular dystrophy diagnosis, according to Stadelmann. In petri dish experiments, he and his team have now shown that the correct muscle stem cells are equally capable of fiddling together and forming young muscle fibers.

The first clinical trial will include five to seven patients with muscular dystrophy before the end of the year, according to Spuler. But there is still an option for individuals who are in wheelchairs to walk and play in wheelchairs. However, the concept of repairing larger muscles, such as those that are required for standing and walking, is already under consideration. However, the molecular tools would have to be so effective that they can be introduced without any reservations not only into isolated muscle stem cells, but also into the degenerated muscle.

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