Genetic Engineering & Biotechnology News

OCT15 2017

Genetic Engineering & Biotechnology News (GEN) is the world's most widely read biotech publication. It provides the R&D community with critical information on the tools, technologies, and trends that drive the biotech industry.

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Genetic Engineering & Biotechnology News | | OCTOBER 15, 2017 | 27 See Animal Models on page 28 These models allow researchers to study the development, progression, and metastat- ic behavior of tumors, beginning at an ani- mal's birth. The models are used to identify predictive blood biomarkers, characterize different types of tumors, search for thera- peutic targets, and study the safety and ef- fectiveness of experimental treatments. For NF1, Surrogen has shown that the ap- pearance and progression of the disease in hu- mans, which typically develops before puberty, is mirrored in the model pigs. Figure 2 shows a young pig with hyper-pigmented café-au-lait spots on its skin, which are characteristic of NF1 and under which deep nerve tumors of- ten develop in both humans and pigs. Advances in gene-editing technology are making it easier for companies such as Sur- rogen to progress from making single muta- tions in a genome to creating animal models with multiple mutations, which is especially important for studying cancer. Surrogen is also creating increasingly humanized swine models by introducing entire human genes. These models facilitate studies of monoclonal antibody-based immunotherapies. Also in de- velopment are inducible, organ-specific swine models, such as animals in which researchers can activate oncogenes known to induce pan- creatic cancer only in pancreatic cells, or turn off tumor suppressor genes only in certain ar- eas of the brain to induce glioblastoma. Targeting T Cells and Natural Killer Cells Preclinical immuno-oncology research in animal models requires human immune cells that are fully functional. "You need to be able to study human proteins on appropriate immune-cell targets in a preclinical setting" and to widen the possibility for detecting both on-target and off-target effects, says Azusa Tanaka, Ph.D., product manager at Taconic Biosciences. Taconic uses CRISPR/ Cas9, knockout/knockin, and transgenic technologies to create genetically engineered mice and rats, and offers a super-immuno- deficient model portfolio that includes the CIEA NOG mouse. The NOG portfolio in- cludes genetically humanized mice express- © 2017 MaxCyte, Inc. All Rights Reserved. Accelerating Your Biotherapeutic Discovery, Development and Biomanufacturing From concept to care, our delivery platform for cell engineering helps you unleash the power of the cell. • Scalable transfection systems and scientific support to ensure your success • Delivery platform for CHO-based transient and stable protein production • The efficiency and viability you need from bench to clinic • Use any cell to identify the right candidate faster • Milligram to grams of protein from a single transfection • Trusted by 9 of the top 10 global pharmaceutical companies Any Cell. Any Molecule. Any Scale. ® Unleash your power: Translational Medicine Figure 2. This young pig, developed as a model for studying neurofibromatosis type 1 (NF1) using genome editing and somatic cell nuclear transfer, exhibits a café-au-lait spot characteristically seen in human patients with the NF1 gene mutation. Surrogen Figure 3. Taconic uses a variety of genome -editing and transgenic technologies to produce a por tfolio of super- immunodeficient NOG mice that are genetically engineered to express human cytokines and cells humanized through modifications to hematopoietic stem cells. Taconic

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