A team of Chinese researchers have created an efficient and very safe technology for editing RNA that could avoid the side effects to a great extent and also the ethical concerns which came from the previous technologies of gene-editing. The study has been published in the journal Nature Biotechnology.
This feat comes after half a year when Chinese researcher He Jiankui claimed that he had made the world’s first gene-edited twins who are immune to HIV. This announcement caused a whirlwind of condemnation all over the world. Many Chinese and international researchers condemned it as any application of gene editing was unethical if used on human embryos for reproductive purposes. He used the technology of CRISPR-Cas9, that was adapted from a genome editing system which occurred naturally in bacteria. The Cas9 enzyme, a protein that plays a very important role in the immunological defence of specific bacteria against the DNA viruses would be introduced for cutting the DNA of the viruses in the human body.
Wei Wensheng, a leading researcher of the technology and also a biologist at the Peking University said that this technology essentially depends on the delivery of chemically modified guide RNAs or exogenous proteins that may lead to a delivery barrier.
Zhou Zhuo, another research team member said that on the contrary, the latest technology known as Leveraging Endogenous ADAR for Programmable Editing of RNA or LEAPER uses the native proteins and hence does not alter the DNA in a direct manner. Because of these factors, it would not bring any heritable changes and is safe. It uses engineered RNA’s for recruiting native enzymes to change certain adenosine to inosine. Zhou also stated that experiments conducted at a cellular level in the last two years showed that LEAPER is capable of achieving editing efficiencies up to 80 percent. He further mentioned that the team is now conducting tests on rats. Hence we have to wait to understand if it is suitable for use in human beings.
LEAPER is active in a very broad spectrum of cell types which includes several human primary cell types. It can also restore the deficient cells of patients having Hurler syndrome without evoking any kind of innate immune responses. Being a single-molecule system, LEAPER ensures highly precise and efficient RNA editing with large scale applications for basic research and therapy.
Journal Reference: Nature Biotechnology