enGENEering our Future: A Multi-sector Endeavor
October 12th, 2023 @ Texas A&M Annenberg Presidential Conference Center
Speakers
University of Michigan Transgenic Animal Core Facility
Advances in Biomedical Research through CRISPR/Cas9 Genome Editing
Thom Saunders is the Director Emeritus of the University of Michigan Transgenic Animal Core Facility and Professor Emeritus of Genetic Medicine at the University of Michigan. He currently serves as Vice President of the International Society for Transgenic Technologies. Under his leadership the Transgenic Core established an outstanding track record in producing genetically engineered mice, rats, and mouse embryonic stem cells. The Transgenic Core CRISPR/Cas9 Pipeline has delivered thousands of gene edited mice and rats to research labs. The animals carry modifications that ablate gene expression, genes with point mutations, reporter insertions, or conditional (floxed) genes. They are used to model human diseases and to explore gene function. CRISPR/Cas9 gene targeting has superseded methods that rely on random transgene integration and ES cell technology. The efficiency of transgenic mouse and rat model production in the Transgenic Core sets or exceeds standards in the published literature. In addition to precision gene edited animal models he collaborated with investigators to produce over 20,000 transgenic mouse and 1000 transgenic rat founders by the random integration of transgenes in the genome. He is a recognized authority nationally and internationally. His recently published compilation of gene editing methods for animal models is titled “Transgenesis: Methods and Protocols” (https://doi.org/10.1007/978-1-0716-2990-1). Precise gene targeting made possible by CRISPR-Cas9 genome editing will advance biomedical research and our understanding of disease mechanisms.
University of Texas at Austin
Turning Bugs into Features: Insect Paratransgenesis and Symbiont-Mediated RNAi
Many insects have co-evolved associations with microbial symbionts that are more consequential than our relationship with the human microbiome. These symbionts may live within insect cells, be inherited across generations, and support host survival. My research group and our collaborators have developed genetic toolkits for engineering diverse bacterial symbionts associated with flies, aphids, bees, and other insects. Because of their important and integrated functions, engineered symbionts can be used to study insect biology, protect beneficial insects, and prevent pests from vectoring disease. Recently, we have examined how culturable “protosymbiont” strains of Serratia symbiotica colonize aphids and demonstrated that they are transmitted to offspring. We are now attempting to attenuate the pathogenicity of these strains to initiate new, stable symbioses. In other work, we have shown that the honey bee gut symbiont Snodgrassella alvi can be engineered to continuously express double-stranded RNAs that induce a targeted RNA interference (RNAi) response in their hosts. These engineered symbionts can be used to knock down expression of bee genes as a tool for functional genomics. To protect pollinator health, we have also used double-stranded RNA expression by symbionts to prime the bee immune system against viral infection and induce a self-killing response in parasitic Varroa mites feeding on bees.
Solid Biosciences
From Dr. Burg:
“I work for Solid Biosciences (Home • Solid Biosciences). We are a gene therapy company using AAVs to create targeted treatments for rare genetic disorders. Our primary candidate is for treatment of Duchenne Muscular Dystrophy. Our potential drug products may have the ability to not just treat these diseases but to cure people. For them to work, the foundation of the drug must be encoded to meet the missing or mutant gene while being able to reach the targeted cell. Therefore, not only do we need to worry about the gene for expression but must consider the packaging (type of AAV) and the method of production and purification to be able to deliver a quality drug product.”
Connecticut Agricultural Station
University of Connecticut
Small Things Considered: Using RNA Molecules and Nanotechnology to Control Plant Pathogens
Plant viral diseases cause over $30 billion in global crop losses annually. Because there are no antiviral treatments, plant viral diseases are extremely hard to control, and farmers must choose resistant plant varieties or spray pesticides to control virus-carrying insects. The da Silva Lab group is working on developing antivirus therapeutics composed of target dsRNA molecules. The trouble is that the effectiveness of applying “naked” dsRNA on leaves is short-lived – dsRNA is quickly assimilated by plant defense mechanisms and is also degraded by environmental factors. In his seminar, Dr. da Silva will showcase the latest research from his group in synthesizing and characterizing different nanocarriers to protect dsRNAs from degradation to prolong their “vaccination effect” against plant viruses. He will also discuss the prospect of transferring this technology to managing other plant pathogens and using nanocarriers to deliver CRISPR/CAS Systems for gene editing.
