核酸科学是当前国际生物医学领域的前沿和热门研究方向，发展迅速，对推动精准医学研究和人类健康起到重要作用。为了推动国内核酸科学和产业的整体发展，推动国内与全球核酸科学研究领域的整合，我们已经成功发起举办四届广州核酸国际论坛。历届论坛演讲嘉宾均由诺贝尔奖得主领衔，汇聚全球核酸领域知名专家学者，议题涵盖医疗、科研及工业等领域的研究热点和最新进展，旨在为中外科学家提供一个高端交流平台，共同分享和探讨本领域的最新研究成果、应用前景及产业化方向。论坛的知名度和影响力得到广大参会人员的高度认可，被誉为核酸研究领域学术风向标。 2017年我们将迎来第五届广州核酸国际论坛的召开，本届论坛将邀请世界顶尖科学家及知名企业家齐聚一堂，与您分享本领域的最新成果和进展。本届论坛主题如下：

1、非编码RNA国际研究前沿

2、核酸诊断与标志物发现

3、核酸药物临床试验

4、核酸治疗与药物开发

Uttiya Basu received his Ph.D. degree in Molecular Biology from Albert Einstein College of Medicine in 2004. After finishing postdoctoral training in the laboratory of Dr. Frederick Alt at Harvard Medical School in 2009, he joined the faculty of Columbia University, Department of Microbiology and Immunology. His laboratory utilizes technologies Involving genome engineering, genomics and biochemistry. His research is focused on the various mechanisms by which non-coding RNA transcription controls genome architecture in pluripotent and differentiated mammalian cells. He has been awarded the NIH director’s New Innovators Award, the Leukemia Lymphoma Society Career Development award, The Pershing Square Sohn Cancer Research Alliance prize, Irvington Institute and Cancer research institute post-doctoral fellowship and Lymphoma Research Foundation Award for cancer research. 

Vadim Y Dudkin is Scientific Director and head of Centyrex targeted delivery team at Janssen, focusing on solving intracellular and tissue specific delivery of nucleic acids, small molecule drugs, and other payloads. Vadim has 16 years of experience in nucleic acids therapeutics, bioconjugates, and small molecule discovery research at Janssen, Merck, and Memorial Sloan-Kettering. Prior to joining Janssen, Vadim led a team of scientists engaged in siRNA delivery at Merck and Co.  

Torsten Hoffmann joined Silence Therapeutics as Chief Operating Officer in June 2017. He has over 20 years of international R&D management experience. Prior to joining Silence, Torsten served as Chief Scientific Officer and Managing Director at Proteros biostructures from July 2015 and before then as Chief Scientific Officer and Executive Vice President at Zealand Pharma from July 2013. He previously spent 16 years at Roche in roles of increasing responsibility up to Site Head of Medicinal Chemistry at the company head quarter in Switzerland, and Head of the Global Roche Postdoc fellowship program. Torsten is the lead inventor of the anti-emetic medicine Netupitant, discovered at Roche and approved by the FDA as Akynzeo© in 2014. Torsten holds a PhD in Chemistry from the ETH Zürich, Switzerland, which was followed by a role as research associate at the Scripps Research Institute in La Jolla, California. He has authored more than 85 publications, patent applications and published conference reports and has served on a number of advisory boards. His awards include the Feodor Lynen award and a fellowship from the Alexander von Humboldt Foundation, Germany.  

http://www.silence-therapeutics.com/

https://www.linkedin.com/in/hoffmanntorsten/

Dr. Kay is the Head of the Division of Human Gene Therapy and serves as the Associate Chair of Research in Pediatrics. He received a Ph.D. in Developmental Genetics, and M.D. from Case Western Reserve in Cleveland, Ohio. Before coming to Stanford in 1998, Dr. Kay was at the University of Washington as Associate Professor in the Departments of Medicine, Biochemistry and Pathology. Dr. Kay has received many awards. Dr. Kay was on the founding board of directors of the American Society for Gene Therapy and served as the Society’s Vice-President, President-Elect, and President in 2003-2006. He is on Board of Directors of the Oligonucleotide Therapeutics Board of Directors. He was elected to the Association of American Physicians in 2010. Dr.Kay received the American Society for Gene and Cell Therapy’s Outstanding Investigator Award in 2013. He is a scientific founder of Voyager Therapeutics and LogicBio Therapeutics. 

Dr. Kay has published over 250 scientific papers.The focus of the laboratory is to establish the scientific principles required for gene and nucleic acid transfer for the treatment of genetic and acquired diseases. Dr. Kay has worked on the development of many DNA gene transfer vectors and the mechanism by which they transduce tissues in mammals. His group has performed two Phase I/II gene therapy trials for hemophilia B. His laboratory was the first to establish therapeutic RNAi in whole non-embryonic mammals, and RNAi-mediated inhibition of a human viral pathogen (HBV) in animals. His work continues towards defining the molecular limits of delivered and expressed RNAi in vivo as well as the mechanisms involved insi/shRNA-mediated gene silencing and the biological mechanisms involved in miRNA-mediated gene repression. In addition, his laboratory is studying the role that a newly discovered small non-coding RNA plays in mammalian gene regulation. 

Dr. Leberer received his Ph.D. in Biology at the University of Konstanz, Germany (1986). He conducted post-doctoral training in molecular biology at the Banting and Best Institute of the University of Toronto, Canada, and then became a Professor of Biochemistry at the University of Konstanz, Germany (1992). He is currently responsible for R&D Alliance Management at Sanofi, and is the Scientific Managing Director of the Innovative Medicine Initiative COMPACT Consortium on the delivery of biopharmaceuticals across biological barriers and cellular membranes (www.compact-research.org). 

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. He has also served as Head of Biotechnology Germany and a member of the Scientific Review Committee of Aventis Pharma Germany. 

Prior to joining pharmaceutical industry, Dr. Leberer served as Senior Research Officer in genetics and genomics at the Biotechnology Research Institute, National Research Council of Canada, Montreal. His research has focused on the molecular mechanisms of signal transduction and the role of signalling molecules in human diseases. He is the principal 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.

Professor Lilley has worked on the structure and dynamics of nucleic acids for more than 35 years. This was initially focused on DNA, and especially the four-way junction – the Holliday junction of genetic recombination. He solved the structure in solution in 1988. In the course of these studies Lilley developed biophysical methods, especially fluorescence resonance energy transfer and more recently X-ray scattering. His FRET study of the four-way DNA junction was really the first in the modern era of such studies, and was very influential. 

Lilley has made many studies of the interactions between DNA junctions and proteins, especially the resolving enzymes. This most recently led to the crystal structure of the eukaryotic enzyme GEN I bound to the product of cleavage of the four-way DNA junction, which has provided significant insight into the recognition process. 

Lilley has had a long-standing interest in the structure and catalytic mechanisms of ribozymes. He has made extensive mechanistic study of the VS and hairpin ribozymes, and in the last three years he has solved high resolution crystal structures of the twister and TS ribozymes.

In RNA structure Lilley has made a detailed study of the structure, folding, protein binding and role of kink-turns, to the point where it is arguably the best-understood structural motif in RNA. Recently this has even gone into the nanotechnology area.

Two guanidine riboswitch structures have recently been solved by X-ray crystallography in the Lilley-lab, and will be discussed in the conference. He has also made a recent study of the structure effect of the common RNA modification N6-methyladenine, and shown how this disrupts trans sheared G•A basepairs, and hence box C/D snoRNP assembly. This will also be addressed in the conference. 

In addition to his research, David Lilley (李大卫) is very active in promotion of links with Chinese science. He is a visiting professor at Xiamen University and at the University of the Chinese Academy of Sciences, a consultant for AB Life in Wuhan and has collaborative links with Shanghai Fudan University. He is also a frequent consultant for the Royal Society on Chinese matters.

Dr. Lin studies the self-renewing mechanism of stem cells in Drosophila, mouse, Hydra and planarian. He also studies germline development and stem cell-related cancers. Dr. Lin received his BS degree from Fudan University (1982), his PhD degree from Cornell University (1990), and his postdoctoral training at the Carnegie Institution for Science. He joined the faculty of Duke University Medical School in 1994, where he rose to Full Professor. He founded and directed the Duke Stem Cell Research Program (2005-2006), and moved to Yale in 2006 to establish the Yale Stem Cell Center. He is also the Founding Dean (Adjunct) of School of Life Science and Technology at ShanghaiTech University (2014- ). Dr. Lin has made key contributions to the demonstration of stem cell asymmetric division and the proof of the stem cell niche theory. He discovered the Argonuate/Piwigene family and their essential function in stem cell self-renewal and germline development. He is also a discoverer of PIWI-interacting RNAs (piRNAs). Recently, he proposed and demonstrated the crucial roles of the Piwi-piRNA pathway in epigenetic programming and in post-transcriptional regulation of mRNA and lncRNA. Dr. Lin has served many leadership roles in the ISSCR, the scientific community, and beyond. Dr. Lin received over 30 awards in his career, and is an Elected Fellow of the AAAS (2010- ).

Additional information about Dr. Lin can be found in the following site:

Web: http://stemcell.yale.edu/people/haifan_lin.profile

A leader in the fields of cellular and developmental biology, Dr. Lodish has isolated, cloned, and characterized numerous proteins and noncoding RNAs that play key roles in formation of blood and fat cells and that regulate metabolism of glucose and fatty acids. His results have important implications for the treatment of anemias, cancer, diabetes, heart disease, and obesity. 

In 1988, the Lodish laboratory identified and cloned the receptor for erythropoietin (Epo), the hormone that controls the production of red blood cells. This led to a lengthy set of ongoing projects on the activation of, and signal transduction by, the erythropoietin receptor in erythroid progenitor cells and the regulation of transcription, apoptosis, and cell division during erythroid development. His laboratory is currently characterizing many novel genes that are important for several stages of erythropoiesis, including chromatin condensation and enucleation. A major focus is determining the roles of many long non-coding RNAs (lncRNAs) that are essential for the differentiation and function of erythroid and myeloid cells, and others essential for formation of white and brown adipose cells. 

Most recently his laboratory developed culture systems for generating mature human red blood cells from hematopoietic stem cells. With Flagship Ventures he cofounded Rubius, a company that uses gene- modified red blood cells as vehicles for the long- term introduction of many novel therapeutics, immunomodulatory agents, and diagnostic imaging probes into the human body. 

A Founding Member of Whitehead Institute, Lodish joined the MIT faculty in 1968. He has been a professor of biology since 1976 and professor of biological engineering since 1999. He earned his PhD at Rockefeller University in 1966. He was elected a fellow of the American Association for the Advancement of Science in 1986, a member of the National Academy of Sciences in 1987, and a fellow of the American Academy of Arts and Sciences in 1999. 

Dr. Lodish is a member of the Board of Trustees of Boston Children’s Hospital, where he is also Chair of the Board of Trustees Research Committee. He was Founding Chair of the Scientific Advisory Board of the Massachusetts Life Sciences Center, the group charged with oversight of the state’s 10- year $1 billion investment in the life sciences. He was a founder and scientific advisory board member of several companies including Genzyme, Inc., Arris Pharmaceuticals, Inc, and Millennium Pharmaceuticals, Inc., and has served on the scientific advisory boards of numerous biopharmaceutical companies. 

Dr. Lodish is the lead author of the textbook Molecular Cell Biology. The eighth edition was published in April 2016; the book has been translated into 12 languages. During the 2004 calendar year Dr. Lodish served as President of the American Society for Cell Biology.

Tomas Robert Lindahl FRS FMedSci is a Swedish scientist specializing in DNA damage and repair. He was director of the Clare Hall Laboratories from 1986 to 2005. He was jointly awarded the Nobel Prize for Chemistry in 2015, the Royal Society’s Royal Medal in 2007, the Copley Medal in 2010, and the INSERM Prix Etranger in 2009.

Tomas Lindahl’s Mutagenesis Laboratory at Clare Hall discovered and characterized different DNA repair pathways in a long-term project to provide better understanding of the cellular defence mechanisms against damage to the human genome.

Damaged sites in the chromosomal DNA can result in cell death or cancer, but may be corrected by DNA repair enzymes prior to phenotypic expression. The properties of several nuclear enzymes that remove harmful lesions or local aberrant structures from DNA have been investigated. The absence of such DNA repair factors may result in an increased frequency of malignant transformation or in some cases may be detected as immunological deficiencies.

Dr. Lindahl closed his lab at the Clare Hall laboratories in 2009 but remains an Emeritus Group Leader at the Francis Crick Institute.  

Additional information about Dr. Lindahl can be found in the following sites:

http://en.wikipedia.org/wiki/Tomas_Lindahl

http://vimeo.com/63309327

https://www.britannica.com/biography/Tomas-Lindahl

Dr. Lu started his biopharmaceutical industry career in 1993 and severed as a lab head and senior scientist in Novartis and Digene (Until 2000). Dr. Lu was the co-founder and Executive VP of Intradigm Corporation (2001-2006). Patrick has authored more than 50 scientific papers, review articles and book chapters, and is an inventor for 42 issued and pending international and China patents. He has been an invited speaker in many international conferences throughout the world, and awarded a number of grants from NIH and the State of Maryland governments. Under his leadership, Sirnaomics has developed an enriched pipeline of novel siRNA therapeutic programs and established partnerships with Chinese Pharmaceutical companies and US biotech companies. The first leading siRNA therapeutic candidate developed by Sirnaomics, Cotsiranib®, has entered Phase IIa clinical study in USA, while being approved for entering clinical study in China as 1.1 category of novel chemical drug entity for treatment Hypertrophic Scar condition. Since 2008, Dr. Lu’s commitment for developing novel siRNA therapeutics for unmet clinical needs in China has been well recognized by many awards including “1000 Talents” national program, “Innovation Pioneer” of Jiangsu Province, “Entrepreneur Leadership” of Guangzhou City, etc. He has also led the State 12.5 key program for Novel RNAi Drug Discovery.  

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. 

Additional information about Dr. Mello can be found in the following sites:

http://www.hhmi.org/scientists/craig-c-mello

http://profiles.umassmed.edu/profiles/ProfileDetails.aspx?From=SE&Person=1009

http://www.nobelprize.org/nobel_prizes/medicine/laureates/2006/mello-bio.html

http://en.wikipedia.org/wiki/Craig_Mello#Awards_and_honors

Guillermo Montoya is Professor at the University of Copenhagen (UCPH) and Research Director of the Protein Structure & Function Programme at the Novo Nordisk Foundation Centre for Protein Research (http://www.cpr.ku.dk/). Montoya is also the Chairman of ISBUC, the Integrative Structural Biology Cluster at UCPH (http://isbuc.ku.dk/). In the last years, his main research interests are the structural analysis of macromolecular complexes of the cell cycle such as the mammalian chaperonin CCT complex, where he uses the combination of X-ray crystallography and electron microscopy to gain insight about their working mechanisms. Dr. Montoya is also systematically pursuing the structure-function analysis of endonucleases, which are of great interest because of their potential applications in gene therapy. He is member of international project evaluation panels and also reviews projects for European funding agencies (ERC, EU, SNF, DFG, NOW, MINECO, Welcome Trust) and serves as an associate editor and ad-hoc reviewer for high-profile journals. 

Dr. Motamedi is an Assistant Professor in Medicine at Harvard Medical School and Massachusetts General Hospital Cancer Center. His group is interested in understanding the molecular mechanisms by which chromatin and non-coding RNAs initiate, maintain and propagate epigenetic states in eukaryotes. He received his Ph.D. from University of Alberta in Canada, and joined the Danesh Moazed lab at Harvard Medical School for his postdoctoral training initially as an NSERC and later as a CIHR postdoctoral fellow. His work as a postdoctoral trainee led to the proposal of the Nascent Transcript Model in which long noncoding RNAs provide a platform for the assembly of chromatin proteins in cis relative to their site of synthesis. His lab is now interested in uncovering the mechanisms by which eukaryotic cells establish the transcriptional program of quiescent cells. He has received several awards including the V Scholar and V Scholar Plus and served as reviewer for several high-profile journals and international granting bodies. 

Dr. Richard J. Roberts is the Chief Scientific Officer at New England Biolabs, Beverly, Massachusetts. He received a Ph.D. in Organic Chemistry in 1968 from Sheffield University and then moved as a postdoctoral fellow to Harvard. From 1972 to 1992, he worked at Cold Spring Harbor Laboratory, eventually becoming Assistant Director for Research under Dr. J.D. Watson. He began work on the newly discovered Type II restriction enzymes in 1972 and these enzymes have been a major research theme. Studies of transcription in Adenovirus-2 led to the discovery of split genes and mRNA splicing in 1977, for which he received the Nobel Prize in Medicine in 1993. During the sequencing of the Adenovirus-2 genome computational tools became essential and his laboratory pioneered the application of computers in this area. DNA methyltransferases, as components of restriction-modification systems are also of active interest and the first crystal structures for the HhaI methyltransferase led to the discovery of base flipping. Bioinformatic studies of microbial genomes to find new restriction systems are a major research focus as is the elucidation of DNA methyltransferase recognition sequences using SMRT sequencing and a new approach to m5C containing recognition sequences. 

Yuanxin Xu joined Alnylam Pharmaceuticals in April 2015 as Sr. Director managing Bioanalytical Sciences group. Her responsibilities include dmanagement of method development/validation and sample analysis (done by Alnylam labs and CROs) to support RNAi therapeutic development from pre-clinical to clinical stages. Multiple platforms (such as mass spec, HPLC, qPCR, LBA, and clinical IVD methods) have been in use to evaluate drug mechanism of action, PK/TK/metabolites, PD/safety biomarkers, and immunogenicity (ADA), etc. 

From 2002-2015, she was Pr. Scientist to Sr. Scientific Director at Genzyme/Sanofi, her responsibilities included clinical assay development/validation and supporting clinical sample analysis for drug development and NDA/BLA filings (protein and small molecule drugs, cell/gene therapies, peptides and an antisense oligonucleotide). She was also Scientific Liaison for Sanofi-CRO strategic alliance. Prior to Genzyme, she studied transplantation and induction of immune tolerance at BioTransplant. 

Yuanxin received her Ph.D. in Biochemistry (1990) from Iowa State University in Biochemistry as a CUSBEA scholar (China-US government program), and Bachelor of Medicine (1984) from Beijing Medical University (now Peking University). She is an active contributor to AAPS LBABFG, journal reviewer, and invited speaker.

Dr. Yang received his BS degree in Microbiology from FuDan University in 1995 and PhD degree in Biochemistry and Molecular Biology from Shanghai Institute of Materia Medica/Shanghai Research Center of Biotechnology, CAS, under the supervision of Professor Shengli Yang, in 2000. He performed his postdoctoral research in International Agency for Research on Cancer-IARC under the supervision of Professor Zhao-Qi Wang (2000-2005) and Clare Hall Laboratories, Cancer Research UK under the supervision of Professor Tomas Lindahl (2005-2008). He was awarded NSFC Distinguished Young Scholars and MOST Youth Science & Technology Innovation Leader. His research interests mainly focus on dissecting the regulatory roles and biological significance of epitranscriptomic RNA modifications. He has contributed in a breakthrough point of expanding new research frontiers of RNA methylation-mediated epitranscriptomics by identifying mammalian RNA methyltransferases for N6-Methyladenosine (m6A) and 5-Methylcytosine (m5C) and demethylases for m6A, and regulatory roles of m6A and m5C in RNA processing.