Researchers from Tomsk Polytechnic University found that polymer and hybrid silica-coated micro-capsules are more efficient in genome-editing when applying CRISPR-Cas9. In the future, this development will significantly simplify and increase the efficiency of genome editing. Which can treat previously uncured inherited diseases, such as Alzheimer’s, hemophilia and many others.
Clustered regulatory interspaced short palindromic repeats / Cas9 (CRISPR-Cas9) is a revolutionary genome-editing technology with huge potential for research and clinical applications. According to Prof. Boris Fehse of the University Medical Center Hamburg–Eppendorf, bacteria have an adaptive immune system ensuring recognition and eradication of viruses if they try to infect them more than once.
For this purpose, bacteria incorporate short sequences of the viral genome into their own genome and utilize them as a template to synthesize short complementary RNAs that recognize a phage genome using the key-lock principle.
CRISPR-Cas 9 procedures present a safety challenge. These delivery methods are highly toxic. When they used, some of the edited cells die. To solve this problem, the researchers decided to use 2-2.5 micrometer polymeric and hybrid silica-coated capsules (SiO2).
They loaded with genetic material and delivered to the target cell. It absorbs them through micropinocytosis, a process in which cells capture relatively big particles. Thus, microcapsules enter the cytoplasm of cells and release their contents. The shell itself gradually dissolves.
Researchers say, delivery efficiency with these microcapsules is achieved due to low toxicity. The study showed that over 90% cells survive after transfection. Thus, the percentage of edited cells became significantly higher in comparison when liposomes used for transfection.
The results allow the scientists to conclude that the microcapsules represent promising vectors for application of genome-editing tools. The next stage, according to the researchers, will be the application of CRISPR-Cas9 using microcapsules to deliver genetic material in vivo studies.
More information: [nanomedicine]