CRISPR and Alternative Approaches – a virtual mini workshop at Wiley

Friday, September 11 – 11:00 AM – 5:00 PM (ET)

Theme: Since the invention of CRISPR-CAS9 gene targeting technology, much effort has been made in resolving the issues of off-target modifications, multiple loci targeting, base-editing, and genome-wide screens for regulatory components and drug targets. The abundance of new developments and applications can be overwhelming. In this workshop, we bring scientists together to discuss CRISPR technology, its potential and limitations, and alternative versions or approaches that are cheaper and/or safer

1. The current status of CRISPR technology, its technical and application potential and limitations 2. Alternative versions or approaches that are better controlled and safer than CRISPR

Organizer: Alison Liu, Editor, Advanced ([email protected])

Sponsors: Advanced Genetics & Cell & Molecular Biology Portfolio at Wiley. Advanced Genetics is a newly launched Open Access journal at Wiley, which aims to publish the best genetic and genomic research in all areas (https://onlinelibrary.wiley.com/journal/26416573).

Presenters: We have invited 6 scientists to give a talk at this half-day workshop. The presenters have options to record and share their presentations with participants and/or exhibit them on the website of Advanced Genetics.

Audience: Scientists who are developing or are interested in developing CRISPR technology, alternative versions or approaches of CRISPR, and their applications.

Format: Presenters and audience will be provided with a Microsoft Teams link and instructions a week in advance.

Cost: Free for attendance

PROGRAM (see the next page)

PROGRAM Friday, September 11 – 11:00 AM – 5:00 PM (ET)

11:00 am Introduction Alison Liu, Editor, Advanced Genetics, Wiley publishing

11:05 am Inducing DSBs in plants: from gene editing to chromosome engineering VIDEO Holger Puchta, Karlsruhe Institute of Technology, Germany

12:05 pm Engineering for tolerance to environmental stress and enhanced nutritional content Pamela Ronald, University of California, Davis, California

Break

1:20 pm Better than CRISPR VIDEO Keynote Speaker: George Church, Harvard University, Boston, Massachusetts

2:35 pm Non-editing CRISPR: modulating gene expression to treat genetic disorders VIDEO Navneet Matharu, UCSF, San Francisco, California

3:15 pm Identifying drug-resistant mutations using CRISPR-mediated base editing VIDEO Gaelen Hess, , Stanford, California

3:55 pm CRISPR diagnostics for the detection of SARS-CoV-2 James Broughton, Mammoth Biosciences, South San Francisco, California

SPEAKER BIOGRAPHY (see the next page)

George M. Church, PhD ’84, is professor of genetics at Harvard Medical School, a founding member of the Wyss Institute, and director of PersonalGenomes.org, the world’s only open-access information on human genomic, environmental, and trait data. Church is known for pioneering the fields of personal genomics and synthetic biology. He developed the first methods for the first genome sequence & dramatic cost reductions since then (down from $3 billion to $600), contributing to nearly all “next generation sequencing” methods and companies. His team invented CRISPR for human stem cell genome editing and other synthetic biology technologies and applications – including new ways to create organs for transplantation, gene therapies for aging reversal, and gene drives to eliminate Lyme Disease and Malaria. Church is director of IARPA & NIH BRAIN Projects and National Institutes of Health Center for Excellence in Genomic Science. He has co-authored more than 515 papers and 130 patent publications, and one book, “Regenesis”. His honors include Franklin Bower Laureate for Achievement in Science, the Time 100, and election to the National Academies of Sciences and Engineering.

Dr. Pamela Ronald is a Professor in the Dept of Plant Pathology and the Genome Center, UC Davis. She completed her Ph.D. at UC Berkeley (1990), earned a B.S. from the Reed College (1982), an M.S. from Stanford University and an M.S. from the University of Uppsala, Sweden. Ronald uses genetic techniques to understand the plant response to infection and tolerance to environmental stress. With her collaborators, she received the 2008 USDA National Research Initiative Discovery Award and the 2012 Tech Award for the innovative use of technology to benefit humanity. Ronald’s book, "Tomorrow’s Table" was selected as an influential book with the power to inspire college readers to change the world. Her 2015 TED talk has been viewed by more than 1.8 million people. In 2019, she received the ASPB Leadership Award, an honorary doctorate from the Swedish Agricultural University and was elected to the National Academy of Sciences.

Dr. Holger Puchta is director of the Botanical Institute and holds since 2002 the Chair of Plant Molecular Biology and Biochemistry at the Karlsruhe Institute of Technology (KIT) in Germany. After his study of biochemistry at the University of Tübingen and his PhD at the Max-Planck-Institute for Biochemistry in Munich he joined the laboratory of Barbara Hohn at the Friedrich Miescher Institute in Basel, Switzerland before he became in 1995 group leader at the Leibnitz Institute for Plant Genetics in Gatersleben (IPK). In 2000 he obtained his habitation in genetics from the University Halle. He was worldwide the first scientist to demonstrate that site-specific nucleases can be applied to induce different kinds of controlled change in plant genomes. His group elucidated major mechanisms of DNA double strand break repair and he was one of leading scientist adopting the CRISPR/Cas technology to plants. For his work on plant genome engineering, he was nominated “Pioneer of Plant Biotechnology” by the Plant Biotechnology Journal and he awarded twice with an advanced grant of the European Research Council. A recent focus of his research centers around CRISPR/Cas mediated plant chromosome engineering. Dr. Navneet Matharu is an Assistant Adjunct Professor and Associate Researcher in the Department of Bioengineering and Therapeutic Sciences, Institute for Human Genetics at the University of California, San Francisco (UCSF). She is a principal investigator at Innovative Genomics Institute. Matharu is the winner of the Charles J. Epstein Trainee Award for Excellence in Human Genetics Research 2017, American Society of Human Genetics. During her PhD in molecular biology and developmental genetics at CCMB, India, Matharu discovered the mammalian homologue of GAGA Factor/ThPOK and show that the T-cell differentiation factor is an important chromatin regulator of homeotic gene complex. She joined Dr. Nadav Ahituv group as a postdoc and through her research at UCSF, she seeded an innovative approach and exemplified a proof of concept study to show how cis-regulatory elements like enhancers could be utilized as therapeutic targets for CRISPR/dCas9 based gene modulation to treat dosage sensitive diseases. Matharu has pioneered the use of non-editing form of CRISPR-mediated-activation as a blueprint for treating haploinsufficient genetic disorders. She is an enthusiastic communicator, winner of the People’s Choice Award in the nation’s first-ever postdoc slam competition at UCSF and actively engages with rare- disease community and patient advocacy group.

Dr. Gaelen Hess is a post-doc in the lab of Michael Bassik at Stanford University and will be starting his own independent lab at UW-Madison. He has employed high-throughput screening platforms to study the functions of genes, SNPs, and protein variants in mammalian cells. Specifically, he has developed the novel genome-editing technology, CRISPR-X, which performs targeted diversification at endogenous loci using the somatic hypermutation machinery. This targeted diversification via point mutations enables the identification and functional annotation of genetic variants. He has applied this base-editing technology to investigate mechanisms of drug resistance and validate drug target predictions.

Dr. James Broughton is Research Lead at Mammoth Biosciences, where he is responsible for driving the development of new CRISPR diagnostic technologies and assays. James received his doctorate degree at UC San Diego, where he used CRISPR/Cas9 genome editing to investigate the function and specificity of microRNAs in C. elegans. For his postdoc, he joined the lab of Dr. Howard Chang at Stanford University to study the function of RNA modifications on a newly discovered class of RNA, called circular RNAs. After his postdoc, James brought his expertise in RNA biology and CRISPR systems to Mammoth Biosciences. Recently, he was first author on a paper in Nature Biotechnology demonstrating the application of Mammoth’s DETECTR technology to rapidly identify SARS-CoV-2 infections.