Biography:

Guangping Gao received his Bachelor’s degree in Medicine from the West China Medical School of Sichuan University. He completed his PhD training in Molecular Genetics at Florida International University with his work involving the isolation and characterization of the human aspartoacylase gene and the genetic mutations responsible for Canavan disease, a severe form of inherited neurodegenerative diseases. He joined the University of Pennsylvania (UPenn) in 1994 where he has developed his career in Viral Vector Biology for Gene Therapy, served as the Director of Vector Program of Institute for Human Gene Therapy to oversee the vector discovery and development, process development, and vector core and quality control testing.

Abstract:

Since the first proof-of-concept human application in the early 90’s, gene therapy, particularly viral vector-mediated gene therapy, has now entered a stage of unprecedented revolution in clinical translation and commercialization. Hundreds of clinical trials have been done or are under way, hundreds of gene therapy companies established and went to IPO worldwide. To date three gene therapy drugs have been commercialized: Two in China and one in Europe, and several others are in the process of final approval by FDA in the U.S. Gene therapy can be accomplished through in vivo and ex vivo approaches by gene replacement for loss-of-function genetic diseases, gene silencing for gain-of-function genetic disorders, gene editing for any genetic diseases and gene addition for treating acquired infectious diseases or cancer. A major challenge in gene therapy is how to efficiently and safely deliver the therapeutic gene to the tissue and cell types needed and make it work as long as possible, ideally accomplishing lifetime gene correction by a single dose. The vehicle to deliver the gene payload, called vector, is the key element for gene therapy. The progress of human gene therapy in the past decades has been primarily driven by vector development. Among all different vectors available for gene therapy to date, adeno-associated virus (AAV)–based vector stands out for its high efficiency, stability, and low immunogenicity/toxicity, holding great promise for different gene therapy applications. AAV is a common benign residential virus that can persist in primate tissues for life time without integrating into host genomes and causing disease. This presentation will review the key principles, history, current status, main challenges and future directions of human gene therapy. The presentation will showcase discovery and development of novel AAV vectors and examples of AAV gene therapy development for treating inborn metabolic errors, neurodegenerative diseases, infectious diseases and cancer by gene replacement, somatic in vivo gene editing, gene silencing and gene addition therapy as well as using rAAV for simple and robust embryonic genome editing to create rodent and nonhuman primate animal models of human diseases.

Biography:

John D Loike, PhD serves as Director for Special Programs for the Center for Bioethics and is the Co-Director for Graduate Studies in the Department of Physiology and Cellular Biophysics at Columbia University College of Physicians and Surgeons. He is also an Advisory Board Member of the Columbia University Center for the Study of Science and Religion, Creator and Faculty Editor of the Columbia University Journal of Bioethics, and Course Instructor for Crossroads in Bioethics, a course for undergraduates offered each spring at Columbia College. His areas of interest in bioethics include stem cells, cloning, neuroethics, bioterrorism, and the interface of science and religion. He has co-organized several national and international conferences on Genetics and Bioethics, and he is the Founding Co-director of BIOCEP (BIOethical Cross-cultural Education Program), a two-week intensive summer internship program designed to promote educational and cultural exchange in medical ethics (medical tourism, emerging infections, stem cell research, reproductive medicine, etc.) with students from Mahidol University in Bangkok, Thailand.

Abstract:

Abstract

New biotechnologies are transforming human life, but are also raising profound bioethical questions. It is critical to assess these bioethical questions before adapting biotechnologies to clinical trials or to commercial applications. Gene editing is one example in which current and future potential medical and scientific benefits and risks of a technology may impact the way society should deal with inherent bioethical challenges. Some ethicists believe that geneticists using CRISPR (clustered regularly interspaced short palindromic repeats)/Cas in germline editing technologies are “playing God” and this may precipitate a backlash from nature that could be disastrous to the human race. There are other ethical questions associated with CRISPR. Will CRISPR lead us down a path to create designer babies? Is it morally legitimate to alter the genome of our children without their autonomous consents? How will eradicating a plant or animal species impact the overall bio-environment? This keynote will highlight critical bioethical issues that every scientist needs to consider when making decisions about translational biotechnologies. The potential benefits of CRISPR can be breathtaking. Currently CRISPR is being tested to reprogram, genetically, human T lymphocytes to kill lung cancer, to correct genetic mutations in patients with sickle cell anemia, and to instruct bacteria to self-destruct, thereby offering a new method to fight antibiotic resistant bacteria. CRISPR could be applied to non-therapeutic situations such as changing hair color, improving human intelligence or enhancing athletic capacity. CRISPR is not just a method to therapeutically edit the genomes of human embryonic cells; it is a powerful, efficient tool for editing genes in any organism. CRISPR can be used to eliminate pathogenic species such as Zika-carrying mosquitoes, to bring extinct animals back to life or modify, genetically, plants that are resistant to infection and drought. Without knowing all of the risk or benefits of CRISPR, a critical bioethical question emerges - how should we proceed in a new era of Promethean overreach to modify the germline in human beings, plants and animals? Balancing the use of new biotechnologies which can improve the quality of life with the introduction of bioethical challenges to the natural process of molecular genetics will be the focus of this critical keynote address.