In the afternoon of September 16, 2019, the RNA and Chromatin Club (RCC) meeting was held in Room A542 of L Building. At this meeting, Dr. Guanglei Li from Prof. Xingxu Huang's research group and Professor Rujuan Liu of School of Life Science and Technology were invited to share their new research results.
During the first talk, Dr. Li showed us his research findings on precise and efficient correction of the pathogenic mutations in human embryos by base editing. Rare genetic diseases account for about 10% of human diseases, and how to cure them is one of the biggest challenges in current medicine. At present gene therapy method has been increasingly used in medicine, however, it still has many disadvantages, such as cost, efficiency and delivery. Dr. Li proposed that base editing in human embryos might be a good strategy for correction of pathogenic mutations in humans. A mutation in Marfan Syndrome was corrected in most tested human embryos with very high efficiency by this method. By deep sequencing, they also found very few changes at the potential off-target sites. Therefore, it indeed seems to work better than the previous CRISPR-based gene therapy methods. This study was published on Molecular therapy and considered as “the most successful attempt to gene edit human embryos so far.” (https://singularityhub.com/2018/08/28/new-study-is-the-most-successful-attempt-to-gene-edit-human-embryos-so-far). This work was also highlighted by Science, MIT Technology Review, etc., and was selected as the first of Best of 2018 in Molecular Therapy of American Society of Gene and Cell Therapy.
For the second half of the meeting, Professor Liu gave a talk entitled “A novel tRNA modification enzyme plays roles in both transcription and translation”. RNA modification occurs in all living organisms, and is one of the most evolutionarily conserved properties of RNAs. Among all RNAs, tRNA is the most heavily modified type of RNA. Modifications in tRNA play crucial roles in maintaining translation efficiency and have been linked to human diseases. Professor Liu's lab identified a novel tRNA modification enzyme TrmX. It can modify five types of tRNA as a methyltransferase. Prof. Liu also found that TrmX can bind to DNA and interact with other factors in transcription. Thus, Prof. Liu hypothesizes that TrmX plays important roles in both transcription and translation.
RCC is aimed to provide a platform for the students, professors and other research scientists in School of Life Sciences at ShanghaiTech University to share their recent experimental results and exchange ideas. It has been successfully held many times in our school. Two speakers are invited to share their research results at each meeting. We encourage more junior investigators to participate in RCC so that they will gain an experience to talk about their work in front of a large audience.