Scientists were able to edit the genome in different tissues and organs

Scientists from the St. Petersburg Polytechnic University took part in the study

Velikogo together with scientists from St. Petersburg State Medical University named after.Pavlova, ITMO University and the University of Hamburg. The goal of the scientists’ work was to examine the delivery of genome editing tools to organs and tissues and compare their development with existing analogues. 

Researchers have developed a polymer carrier,possessing a number of unique properties. Genetic material of various sizes and structures can be loaded into the “universal containers” of its structure: from small interfering RNAs to large messenger RNAs. Scientists have demonstrated the effectiveness of such delivery on human stem cells.

CRISPR / Cas9 technology works on the principle"Molecular scissors", which are important to properly deliver the cells that contribute to the development of the disease. The complex binds to the desired region of the genome, in which the Cas9 protein cleaves DNA.

It's kind of like a flash drive with information.which, when entering the body, forces cells to perform certain actions, for example, training the immune system to fight “enemy” proteins of the virus. Typically in medical applications, carriers are used to deliver specific genetic materials, but we have proven that we are capable of delivering genetic materials of different sizes. This technology opens up new horizons for the development of non-viral delivery systems. 

Alexander Timin, Head of the Laboratory of Microencapsulation and Controlled Delivery of Biologically Active Compounds, SPbPU

The researchers compared different delivery methods. They analyzed the efficiency of editing, delivery and toxicity of the media.

Researchers now plan to create medical protocols that will be implemented in medical practice.

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