Nucleic acids science plays a key role in precision medicine, genetic therapy and new solutions for human health. Continuous discovery of new RNA functions and constant emergence of innovative nucleic acids technologies are driving forces for the field and hold enormous promise for the future scientific and technological progress. The China Nucleic Acids Forum (CNAF) led by Nobel Prize winners is an international forum at the forefront of these developments, aims to push forward communications and collaborations in and abroad, and has been successfully held times since 2013. The CNAF has attracted high-profile speakers, including Nobel Prize winners, to highlight recent advances in nucleic-acid based medicine, scientific discoveries, diagnostics and industrial trends. It has been widely recognized as the premier forum in Asia for advancing nucleic acids research and drug development. The 2018 CNAF featured 20+ globally prominent experts discussing the latest advances in nucleic acids research and development.

1. Frontiers in Non-coding RNA Research
2. CRISPR Gene Editing Technology
3. Advances in Oligonucleotide-based Therapeutics
4. Cutting-edge RNA Technologies and Applications

Dr. Mello’s lab uses the nematode C. elegans as a model system to study embryogenesis and gene silencing. His collaborative work with Dr. Andrew Fire led to the discovery of RNA interference (RNAi), for which they shared the 2006 Nobel Prize in Physiology or Medicine. Together they showed that when C. elegans is exposed to double-stranded ribonucleic acid (dsRNA), a molecule that mimics a signature of viral infection, the worm mounts a sequence-specific silencing reaction that interferes with the expression of cognate cellular RNAs. Using readily produced short synthetic dsRNAs, researchers can now silence any gene inorganisms as diverse as rice and humans. RNAi allows researchers to rapidly “knock out” the expression of specific genes and, thus, to define thebiological functions of those genes. RNAi also provides a potential therapeutic avenue to silence genes that cause or contribute to diseases.Dr. Mello received his BS degree in Biochemistry from Brown University in 1982, and PhD from Harvard University in 1990. From 1990 to 1994, he conducted postdoctoral research at the Fred Hutchinson Cancer Research Center in Seattle, WA. Now Dr. Mello is an Investigator of the Howard Hughes Medical Institute, the Blais University Chair in Molecular Medicine and Co-director of the RNA Therapeutics Institute at the University of Massachusetts Medical School.Besides the Nobel Prize, Dr. Mello’s work was recognized with numerous awards and honors, including the National Academy of Sciences Molecular Biology Award (2003), the Wiley Prize in Biomedical Sciences from Rockefeller University (2003), Brandeis University’s Lewis S. Rosnstiel Award for Distinguished Work in Medical Research (2005), the Gairdner Foundation International Award (2005), the Massry Prize (2005), the Paul Ehrlich and Ludwig Darmstaedter Award (2006), the Dr. Paul Janssen Award for Biomedical Research (2006), the Hope Funds Award of Excellence in Basic Research (2008). He is a member of the National Academy of Sciences, the American Academy of Arts and Sciences, and the American Philosophical Society.

Dr. Südhof received his M.D. degree from the University of Göttingen, Germany, in 1982, after performing an internship in the Göttingen University Hospital. During his medical school training, he worked as an assistant scientist on his doctoral thesis at the Max-Planck-Institut für biophysikalische Chemie in Göttingen from 1978 to 1982, and was award a doctoral degree for this work also in 1982. After completing his medical and scientific training, Dr. Südhof became a postdoctoral fellow in 1983 in the laboratory of Drs. Michael S. Brown and Joseph L. Goldstein at the University of Texas Southwestern Medical School in Dallas, USA, where he cloned the LDL receptor gene and elucidated the cholesterol-dependent regulation of the expression of the LDL receptor. In 1986, Dr. Südhof founded his own laboratory and decided to completely switch fields to work in neuroscience. Over the next two decades, his laboratory first at Southwestern Medical School in Dallas and later at Stanford Medical School in Stanford described the molecular mechanisms underlying the calcium-dependent release of neurotransmitters at a synapse, work for which he was awarded the 2013 Albert Lasker Basic Medical Research Award and the 2013 Nobel Prize in Physiology or Medicine. In the last decade. Dr. Südhof’s work has shifted to a new basic problem in understanding the brain, which is the question of how neurons are wired into circuits via synaptic connections.

Gunter Meister is professor and chair for Biochemistry at the University of Regensburg, Germany. He received his Master degree in Biology (Diploma) in 1999 from the University of Bayreuth, Germany. He obtained his PhD in 2002 from the Max-Planck-Institute of Biochemistry in Martinsried, and the Ludwig Maximilians University in Munich, Germany. From 2003 to 2005 he worked as postdoctoral fellow in the lab of Tom Tuschl at the Rockefeller University in New York, USA. In 2005, he started his own lab at the Max-Planck-Institute of Biochemistry as independent group leader. In 2009, he was appointed full professor and chair for Biochemistry at the University of Regensburg. His research focus is the biochemical analysis of small RNA-guided gene silencing pathways, long non-coding RNAs, RNA binding proteins and RNA modifications in mammals. He contributed to our understanding of the molecular basis of RNAi in human cells and also developed novel silencing approaches. He receives funding from the ERC (staring and consolidator grants) and received the research award of the Engelhorn foundation, the Schering young investigator award and the Bücher medal from FEBS.

Dr. Igor Ulitsky received his PhD in computational biology from Tel Aviv University in 2009, working with Prof. Ron Shamir on algorithms for combination of heterogenous biological data and discovery of functional models. He was then a postdoctoral fellow at Whitehead Institute for Biomedical Research in Cambridge, Massachusetts, working in the lab of Prof. David Bartel on the evolution and roles in embryonic development of long noncoding RNAs (lncRNAs). Since 2013, he is a group leader at the Weizmann Institute’s Department of Biological Regulation. His lab is combining experimental and computational biology to study the evolution, genomics, functions and modes of action of long noncoding RNAs in various mammalian systems, including stem cells, mouse models, and cancer cells. Dr. Ulitsky has been awarded a number of academic honors, including the Wolf Prize for Outstanding PhD Students (2008), the EMBO Long-Term Fellowship for postdoctoral research (2010–2011), and the Heineman Research Award (2018). He is an EMBO Young Investigator from 2016 and recipient of the ERC starting grant (2015). Lab website: https://www.weizmann.ac.il/Biological_Regulation/IgorUlitsky/

Professor  Kay received his  B.S. in physical sciences from Michigan State University, a Ph.D. in Developmental Genetics and M.D. from Case Western Reserve University. Afterwards, Dr. Kay pursued additional medical training and triple board certification in Pediatrics, Medical Genetics, and Inborn Errors of Metabolism at the Baylor College of Medicine. He was appointed to the faculty at the University of Washington in 1993 and moved to Stanford University in 1998.

Professor Kay’s lab made seminal contributions in the field of gene  and nucleic acid based therapeutics. His early work on developing  recombinant adeno-associated viral vectors led to the first clinical trial where such a  vector was administered systemically into humans, and in which he held the original IND.  His lab has made important contributions in the area of miRNA biogenesis, their mechanisms of action, and RNAi therapeutics.  While his lab continues to work in these areas, his group was one of the first to promote the idea that tRNA derived small RNAs (tsRNAs)  were not mere degradation products but rather molecules that play important roles in gene regulation. His lab has shown that one type of tsRNA functions in regulating ribosome biogenesis and is a potential target for treating hepatocellular carcinoma. Professor Kay has published over 250 papers, is currently the deputy editor of Human Gene Therapy, and serves on the editorial boards of other peer-reviewed publications.

Dr. Kay served on the Board of Directors of the Oligonucleotide Therapeutics Society for 10 years. He was on the founding board of the  of the American Society of Gene and Cell Therapy, and served as its President in 2005-2006. He was the  recipient of the society’s Outstanding Investigator Award in 2013.  Dr. Kay received the E. Mead Johnson Award for Research in Pediatrics in 2000. He was elected to the American Society for Clinical Investigation in 1997 and the Association for American Physicians in 2010.  He has organized many national and international conferences, including the first Gordon Conference dedicated to gene therapy.  Professor Kay has and continues to serve on various commercial and academic boards.  He is a scientific co-founder of Voyager Therapeutics, and a co-founder of LogicBio Therapeutics.

Dr. Muthiah (Mano) Manoharan serves as a Senior Vice President, Scientific Advisory Board Member, and a Distinguished Research Scientist at Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA. In 2003, he was the first chemist hired at Alnylam. He and his team pioneered the discovery and development of the chemical modifications that make RNA interference-based human therapeutics possible. This  work led to ONPATTRO (Patisiran), the first RNAi therapeutic approved by FDA in 2018.  Dr. Manoharan has had a distinguished career as a world-leading chemist in the areas of oligonucleotide chemical modifications, conjugation chemistry, and delivery platforms (lipid nanoparticles, polymer conjugates, and complex-forming strategies). Dr. Manoharan and his research group demonstrated for the first time the human therapeutic applications of GalNAc-conjugated oligonucleotides at Alnylam, a platform that has revolutionized the nucleic acid-based therapeutics field with several compounds in the advanced clinical trials.  He is an author of more than 215 publications (nearly 43,000 citations with an h-index of 94 and an i10-index of 367) and over 400 abstracts, as well as an inventor of over 225 issued U.S. patents.  Prior to Alnylam, Dr. Manoharan worked at Ionis (formerly Isis) Pharmaceuticals and Lifecodes Corporation in the field of antisense oligonucleotide therapeutics. He received the M. L. Wolfrom Award from the American Chemical Society Carbohydrate Chemistry Division in 2007 for his contributions to this field. He has been recognized as the Lifetime Achievement Awardee of the Oligonucleotide Therapeutics Society for the year 2019. 

Dr. Leberer received his Ph.D. in Biology at the University of Konstanz, Germany (1986), conducted post-doctoral training in molecular biology at the Banting and Best Institute of the University of Toronto, Canada. He then obtained the Habilitation for Professor of Biochemistry at the University of Konstanz, Germany (1992). From 1989-1998, he served as Senior Research Officer in genetics and genomics at the Biotechnology Research Institute, National Research Council of Canada, Montreal. He was also Adjunct Professor at McGill University, Montreal.

Since joining Hoechst Marion Roussel in 1998, Dr. Leberer carried out various managing roles in this company, Sanofi’s predecessor companies and Sanofi itself, including responsibilities in functional genomics, biological sciences and external innovation for oligonucleotide-based therapeutics. Since 2012, he is Global Alliance Manager for R&D at Sanofi, Frankfurt. In addition, from 2012-2018, he has been the Scientific Managing Director of the Innovative Medicines Initiative COMPACT Consortium on the delivery of biopharmaceuticals across biological barriers and cellular membranes, Brussels.

His research has focused on the molecular mechanisms of signal transduction and the role of signalling molecules in human diseases. He is the co-discoverer of the p21 activated protein kinase (PAK) family of cell signalling proteins and of novel virulence-inducing genes in pathogenic fungi. He is co-author of more than 60 publications in prestigious peer-reviewed journals including Nature and Science.

Dr. Dickinson earned his B.S. in Biochemistry from the University of Maryland, College Park and his Ph.D. in Chemistry from the University of California at Berkeley for work performed with Professor Christopher Chang. His graduate work focused on the synthesis and application of small molecule fluorescent probes for the detection of hydrogen peroxide in living systems. Then, as a Jane Coffin Childs Memorial postdoctoral fellow with Professor David Liu at Harvard University, he developed new methods to rapidly evolve proteins to perform novel functions. Bryan joined the faculty at the University of Chicago in the Department of Chemistry in the Summer of 2014 and was promoted to Associate Professor in 2019. The Dickinson Group employs synthetic organic chemistry, molecular evolution, and protein design to develop molecular technologies to study chemistry in living systems. The group’s current primary research interests include: 1) how lipid modifications on proteins are controlled and regulate cell signaling, 2) developing new evolution technologies to reprogram and control biomolecular interactions, and 3) engineering protein-based systems to understand and exploit epitranscriptomic regulation. The motivating principle of the Dickinson Group is that our ability as chemists to create functional molecules through both rational and evolutionary approaches will lead to new breakthroughs in biology and biotechnology.

Dr. Corey received his B.A. degree from Harvard in 1985 and then conducted his graduate work at University of California Berkeley Chemistry Department under the direction of Dr. Peter Schultz, receiving his Ph.D. in 1990. After postdoctoral work under the direction of Dr. Charles Craik at the University of California San Francisco Dr. Corey began his independent career at the University of Texas Southwestern Medical Center in 1992. Dr. Corey studies how chemically modified nucleic acids can be used to control gene expression. Ongoing project include activation of frataxin protein expression as a potential treatment for Friedreich’s Ataxia, understanding the role of RNAi in mammalian cell nuclei, and understanding the molecular causation and potential treatment of Fuchs’ Dystrophy, a disease caused by mutant trinucleotide repeat RNA.

Rory Johnson’s research program focusses on long noncoding RNAs, their basic biology and their roles in disease, using a combination of bioinformatics and CRISPR-Cas9 genome engineering. Johnson received his MSc in Physics from Imperial College, London, in 2000. He won a Wellcome Trust PhD scholarship at the University of Leeds (UK), where he wrote a thesis on transcriptional and post-transcriptional regulation of gene expression in the mammalian nervous system in 2006. His growing interest in lncRNAs led him first to the Genome Institute of Singapore, then to Roderic Guigo’s lab at the Centre for Genomic Regulation, Barcelona. Here, supported by a Ramon y Cajal fellowship, he worked with the GENCODE annotation project on methods to catalogue lncRNAs in the human and mouse genomes. During this same period, Johnson became interested in the potential of new CRISPR-Cas9 technology to address the great unanswered questions of the lncRNA field: which ones are functional, and which may be targeted in disease? This has been the objective of the Johnson laboratory at the University Hospital of Bern, composed of around 12 researchers, equally split between bioinformaticians and experimentalists. The lab has created a range of CRISPR tools for genome-wide functional screening of lncRNAs in cancer. They are also active members of GENCODE, the International Cancer Genome Consortium and the NCCR ‘RNA & Disease’. Finally, Johnson is involved in Knowledge and Technology Transfer, promoting collaborations between the pharmaceutical industry and NCCR research groups, to accelerate commercialization of RNA biology.

Dr. Gao received his B.S. degree from Nankai University in China (1998), his Ph.D. degree in chemistry from Indiana University, Bloomington (2004), and his postdoctoral training from the Department of Biomedical Engineering at Georgia Tech and the Winship Cancer Institute at Emory University. He has been a faculty member in the Department of Bioengineering at the University of Washington since 2005. His research interests include cancer nanotechnology, molecular engineering, molecular imaging, and drug delivery. He has published >90 peer-reviewed journal articles.

Dr. Dinshaw Patel received his PhD in Chemistry from New York University in 1968 for research in photochemistry. Following postdoctoral training in Biochemistry and Biophysics, he became a joined AT&T Bell Labs and spent the next 15 years undertaking NMR-based studies of the structure and dynamics of cyclic peptides, proteins and nucleic acids. He moved to Columbia University Medical School in 2004 where his group spent the next 8 years doing NMR-based research on DNA mismatches, bulges and junctions, on DNA triplexes and G-quadruplexes, and drug-DNA complexes. He was recruited in 1992 to the Memorial Sloan-Kettering Cancer Center to set up a Structural Biology program. His group’s research initially focused on NMR-based studies of covalent chiral carcinogen-DNA adducts, and complexes of antibiotics and peptides with natural and in vitro selected RNA targets. His laboratory has applied x-ray crystallography to projects on RNA-mediated gene regulation, with subsequent extension to histone-mark and DNA-mark mediated epigenetic regulation, to lipid transfer proteins, to nucleic acid pattern recognition receptors, and more recently cryoEM approaches to study CRISPR-Cas surveillance complexes and their inhibition by anti-CRISPR proteins. His group has complemented their structural efforts with functional studies to deduce mechanistic insights into the biological systems of interest. Dr. Patel is a Member of the National Academy of Sciences and the American Academy of Arts and Sciences.

Dr. Didier Stainier is the director of the Department of Developmental Genetics at the Max Planck Institute for Heart and Lung Research, Bad Nauheim, Germany.  He received his Ph.D. in Biochemistry and Biophysics from Harvard University where he studied the cellular basis of axon guidance and target recognition in the developing mouse brain with Walter Gilbert.  After a postdoc with Mark Fishman at Massachusetts General Hospital (Boston) where he initiated the studies on zebrafish cardiac development, he spent close to 20 years at the University of California San Francisco, expanding his research to investigate questions of cell differentiation, tissue morphogenesis, organ homeostasis and function, as well as organ regeneration, in the zebrafish cardiovascular system and endodermal organs.  In 2012, he moved to the Max Planck Institute where he continues to utilize both forward and reverse genetic approaches to investigate cellular and molecular mechanisms of developmental processes during vertebrate organ formation, in both zebrafish and mouse.  His lab also studies mechanisms of genetic compensation in zebrafish, mouse, C. elegans and yeast.

Dr. Schwartz received his B.Sc. from Tel Aviv University in Israel in 2006, where he then also conducted his PhD work in the field of RNA splicing under the supervision of Prof. Gil Ast, obtaining his PhD in 2010. Dr. Schwartz then conducted his post-doctorate at the Broad Institute of MIT & Harvard under the joint supervision of Aviv Regev and Eric Lander, where his work focused on mRNA modifications, and he established his independent laboratory at the Weizmann Institute in 2015. Dr. Schwartz has pioneered some of the key approaches for systematically detecting and quantifying RNA modifications at a transcriptome-wide scale. His lab bridges experimental and computational approaches, which jointly allowed the development of the first approach for systematically mapping N6-methyladenosine (m6A), pseudouridine, and the first approach for obtaining single-nucleotide resolution mappings of N1-methyladenosine (m1A). His lab aims to unravel the functions and mechanisms of actions through which diverse RNA modification modulate the fate of RNA, and understand how this in turns shapes cellular decision-making.

Dr. Nadav Ahituv is a Professor in the Department of Bioengineering and Therapeutic Sciences and the Institute for Human Genetics at the University of California, San Francisco. He received his PhD in human genetics from Tel-Aviv University working on hereditary hearing loss. He then did his postdoc, specializing in functional genomics, in the Lawrence Berkeley National Laboratory and the DOE Joint Genome Institute. His current work is focused on identifying gene regulatory elements and linking nucleotide variation within them to various phenotypes including morphological differences between species, drug response and human disease. In addition, his lab is developing massively parallel reporter assays (MPRAs) that allow for high-throughput functional characterization of gene regulatory elements and the use of gene regulatory elements as therapeutic targets.

Dr. Gang CHEN received his B.S. degree in Chemistry at the University of Science and Technology of China (USTC) in 2001. He did his Ph.D. studies with Prof. Douglas TURNER in the Department of Chemistry at the University of Rochester. His Ph.D. work involved thermodynamic and NMR studies of RNA internal loops. A better understanding of the sequence dependence of thermodynamics for RNA structures will improve the accuracy of the RNA secondary structure prediction programs such as MFOLD and RNAstructure. He earned his Ph.D. in 2005. He was a postdoctoral fellow in Prof. Ignacio TINOCO’s lab in the Department of Chemistry at the University of California, Berkeley from January 2006 to June 2009. His research in Tinoco lab was on single-molecule mechanical unfolding and folding of RNA pseudoknots by laser optical tweezers, which provided new insights into ribosomal reading-frame regulation by cis-acting mRNA structures. He was a Research Associate in Prof. David MILLAR’s lab in the Department of Molecular Biology at The Scripps Research Institute working on HIV-1 Rev-RRE assembly using single-molecule fluorescence techniques. In July 2010, he joined the faculty in the Division of Chemistry and Biological Chemistry at Nanyang Technological University in Singapore. 

Dr CHEN’s research group in Singapore has been working on probing and targeting RNA structures. By nano-manipulation using optical tweezers in combination with other biophysical methods, the group has revealed at the single-molecule level the contributions of molecular recognition interactions such as a single hydrogen bond in a base pair or base triple to RNA folding and unfolding and characterized the effects of a single proton binding. The lab has unveiled the correlations (i) between mRNA mechanical properties and mRNA structure induced translational reading frame shifting and (ii) between mechanical properties of tau pre-mRNA splice site hairpin structures and pre-mRNA alternative splicing activities. In addition, the lab is developing a molecular recognition platform based on chemically modified Peptide Nucleic Acids (PNAs) for the targeting of RNA structures in a sequence- and structure-specific manner. Specifically, the lab programs the dsRNA-binding PNAs (dbPNAs) with a new four-letter chemical code (T, L or R, E or S, and Q) for the recognition of RNA Watson–Crick duplexes, through the formation of major-groove triples. The lab has demonstrated that short (e.g., 10-mer) PNAs containing L, R, E, S, or Q show enhanced sequence-specific recognition of RNA base pairs in dsRNAs with significantly weakened binding to ssRNAs or dsDNAs at near-physiological conditions. The cell culture studies show that PNAs conjugated with small molecules are bioactive in the targeting of viral and cellular RNAs. The lab is further developing the dbPNAs platform as useful diagnostic tools and therapeutic drugs to fight against viral infections, cancers, and neurodegenerative diseases.