RNA Therapeutics in Human Diseases

Professor Phei Er Saw is a prominent figure in the field of biotechnology and nanomedicine, currently serving at the Sun Yat-sen Memorial Hospital of Sun Yat-sen University. Her distinguished career is marked by significant contributions to the development of innovative cancer therapies using nanoparticle technology. Professor Saw's academic journey began at the University of Malaysia Sabah, where she earned a Bachelor of Biotechnology with honors in 2008. Her undergraduate thesis focused on the analysis of N-glycan and O-glycan uromodulin and its effects in gestational diabetic women. She then advanced her studies at the prestigious Gwangju Institute of Science and Technology and the Korea Advanced Institute of Science and Technology, completing her Ph.D. in 2014. Her doctoral research, which developed lipid-based nanovehicles for cancer therapy, set the stage for her future innovations in the field.Following her Ph.D., Professor Saw undertook a postdoctoral fellowship and later became a senior post-doctoral fellow at Brigham and Women’s Hospital, Harvard Medical School. Here, she deepened her research in nanoparticle technologies, contributing to several high-impact publications. In 2018, she joined the faculty of Sun Yat-sen Memorial Hospital, where she has since been a Professor in the Guangdong Provincial Key Laboratory of Malignant Tumor Epigenetics and Gene Regulation. Professor Saw holds multiple leadership roles, including Director of the Office of Research Integrity and Director of the Manuscript Editing Department at Sun Yat-sen Memorial Hospital. She is also the Vice Director of Research in the Department of General Practice and the Executive Editor of BIO Integration. Her research has earned her a place among the Top 2% Most Cited Scientists in the World in 2023 and other accolades such as the IAAM Young Scientist Award and the USERN Young Scientist Award nomination.Professor Saw's research interests are centered on the development of novel bioactive nanoparticles for targeted cancer therapy. She focuses on siRNA and drug delivery using lipid-based nanoparticles, the formulation of nanoparticle-encapsulated small biologic drug conjugates, nature-inspired nanomaterials for immunomodulation, and tumor-microenvironment responsive nanoparticles. Her work aims to enhance in vivo circulation, deep tissue penetration of small biologics, and provide tumor-specific triggered and effective cancer therapy. With a citation count of over 5,713 and an H-index of 41 as of March 2024, Professor Saw’s contributions to medical research are widely recognized and respected in the scientific community. Her groundbreaking work not only advances the field of nanomedicine but also offers hope for more effective cancer treatments. Professor Phei Er Saw continues to inspire with her dedication to research and innovation, shaping the future of cancer therapy with each discovery and development in the fascinating realm of RNA therapeutics and nanotechnology. Erwei Song is Professor of Breast Surgery at Sun Yat-sen University (SYSU), Member of Chinese Academy of Sciences, “Cheung Kong Scholar" Chair Professor, CMB (China Medical Board) Distinguished Professor and Fellow of the Royal Society of Biology (UK). Currently, he serves as President of Sun Yat-sen Memorial Hospital, SYSU. Erwei obtained his M.D. and Ph.D. at Zhongshan Medical School, and was trained as a surgeon at Sun Yat-sen Memorial Hospital, SYSU. He received postdoc training at Harvard Medical School in 2002 and became an instructor at the CBR Institute of Biomedical Research at Harvard in 2004. His early publication “RNA interference targeting Fas protects mice from fulminant hepatitis” in Nature Medicine Professor Erwei Song, a distinguished academician and a leading expert in RNA therapeutics, has made significant contributions to the field of biomedical research. His remarkable career is marked by extensive research, clinical advancements, and leadership in academic and medical institutions.Born in Guangzhou, China, Professor Song pursued his passion for medicine at Sun Yat-Sen University of Medical Science, obtaining his M.D. in 1993. He continued his studies at the same university, earning an M.S. in Experimental Surgery in 1995 and a Ph.D. in Experimental Hepato-biliary Surgery in 2000. His postgraduate journey took him abroad, where he held postdoctoral fellowships at Essen University Hospital, Germany, and later at the CBR Institute of Biomedical Research, Harvard Medical School. Professor Song returned to China in 2004, starting as an Instructor and Attending Doctor at Sun Yat-sen Memorial Hospital, where he later ascended to the position of Professor and Chief in 2009. His expertise in general surgery and experimental research has led to his holding multiple senior administrative roles, including Vice President and President of Sun Yat-sen Memorial Hospital, Dean of Zhongshan School of Medicine, and Director of the Center for Precision Medicine and the Health Science Center at Sun Yat-sen University.Throughout his career, Professor Song has focused on the application of non-coding RNA in targeted therapy for malignant tumors. His work has not only advanced the understanding of RNA mechanisms in oncology but also led to innovative treatments that have been recognized globally. He is a Fellow of the Hong Kong Academy of Medicine and the Royal Society of Biology, UK, and in 2019, he was elected as a Member of the Chinese Academy of Sciences. His leadership extends beyond academia into healthcare policy and medical ethics, where he has served on various national and international committees, including the Future of Gene Therapy Expert Panel and the NHLBI Working Group of NIH. He has been honored with numerous awards, such as the National Key Talent Project Chair Professor by the Ministry of Education, P.R. China, and the JCA International Award by the Japanese Cancer Association.Professor Song is also prolific in scholarly communication, serving as editor and editorial board member for several esteemed journals, including the Journal of Biological Chemistry, BMC Cancer, and Science China Life Sciences. His editorial contributions ensure that high-quality research reaches a broad audience, fostering a deeper understanding and application of medical sciences. He is actively involved in various professional societies and has significantly influenced medical practices and policies in China. His roles have included Vice Chair of the Biological and Medical Professional Committee of the Young Scientists Association of Guangdong Province and Chair of the Breast Tumor Committee of the Chinese Society of Clinical Oncology. Professor Erwei Song’s career is a testament to his dedication to improving human health through rigorous research, clinical excellence, and public service. His ongoing work continues to inspire new generations of medical professionals and researchers in China and worldwide, advancing the frontiers of medical science and therapeutics.

• Chapter 1 Historical overview of RNA therapeutics.- Chapter 2 RNA: an introduction.- Chapter 3 RNA in the central dogma: mRNA.- Chapter 4 RNA in the central dogma: tRNA.- Chapter 5 non-coding RNAs: long non-coding RNAs.- Chapter 6 non-coding RNAs: microRNAs.- Chapter 7 non-coding RNAs: circular RNAs.- Chapter 8 non-coding RNAs: small interfering RNAs.- Chapter 9 non-coding RNAs: tiRNAs.- Chapter 10 non-coding RNAs: glycanRNAs.- Chapter 11 emerging noncoding RNAs: piRNAs, snoRNAs, xist RNAs.- Chapter 12 extracellular RNAs: biogenenesis.- Chapter 13 extracellular RNAs: functions, crosstalks and therapeutic potential.- Chapter 14 Distinguishing functional roles of protein-coding vs. non-coding RNAs: the possibility of bifunctional RNAs.- Chapter 15 RNA modifications in human diseases.- Chapter 16 RNA screening design.- Chapter 17 single-cell RNA sequencing (sc-RNAseq) in the development of biomedical therapeutics.- Chapter 18 RNA-ligand molecular docking.- Chapter 19 RNA imaging technologies.- Chapter 20 RNA-based imaging system.- Chapter 21 Targeting RNA with small molecules: fundamental principles.- Chapter 22 RNA bio-conjugation: current development in siRNA therapeutics.-Chapter 23 RNA nanotechnology: the fundamentals.- Chapter 24 RNA nanotechnology: biomedical application.- Chapter 25 Current development of RNA delivery systems.- Chapter 26 mRNA vaccines.- Chapter 27 circRNA vaccines.- Chapter 28 Cancer biomarker based on ncRNA liquid biopsy.- Chapter 29 Clinical RNAseq: from molecular basis to clinic.- Chapter 30  Utilization of AI in designing RNA therapeutics.