HUDSON, NY--(Marketwired - Feb 17, 2017) - Taconic Biosciences, a global leader in genetically engineered rodent models and associated services, announced it has generated a novel mouse model of Vici syndrome for use by two investigators studying this rare disease. As a corporate sponsor of the Rare Disease Science Challenge: BeHEARD, hosted by the Rare Genomics Institute, Taconic donated model development services.
Using CRISPR/Cas9 gene editing, Taconic developed and validated a mouse model carrying the EPG5 mutation found in Vici syndrome patients in multiple families. A highly efficient way to generate custom mouse and rat models on a short timeline, CRISPR is one of many technologies Taconic employs to generate models of human disease and support investigator studies of gene function in vivo.
Genetically engineered mice of the Vici syndrome model have been delivered to the investigators working on the BeHEARD project: Mathias Gautel, BHF Chair of Molecular Cardiology at King's College London BHF Research Excellence Centre, and Heinz Jungbluth, Reader in Paediatric Neurology at King's College, and Consultant at the Evelina Children's Hospital, Guy's & St Thomas' NHS Foundation Trust, London.
"The precision of the genetic manipulation by Taconic and their speed of delivery were critical to our work," Prof. Gautel and Dr. Jungbluth said. The investigators are now using the mice to validate the model's replication of the human phenotype of the disease, in terms of neurological, musculoskeletal and other organ symptoms. "If the model replicates the human phenotype with significant fidelity, it could be applied quite rapidly to screening drug compounds for Vici syndrome," Prof. Gautel said.
Rachel and Michael Harris, parents of 10-year-old Vici syndrome patient David, submitted the proposal for the BeHEARD project and are hopeful the model will facilitate development of effective therapies. "While the initial research is focused upon understanding the disease mechanisms, we hope to soon see translational research that will bring new therapies to David and other patients," Michael Harris said.
"Taconic's core mission is to provide animal models that help accelerate drug discovery. Being able to support critical research on a rare disease like Vici syndrome through custom genetically engineered models is extremely rewarding," said Dr. Robert Rosenthal, chief executive officer of Taconic Biosciences.
Taconic has over twenty years' experience designing custom transgenic models for leading pharmaceutical, biotech and academic clients. Taconic is fully licensed to utilize CRISPR/CAS9, homologous recombination and other technologies to develop custom mouse and rat models, including humanized, knockout, knock-in, inducible shRNA, or microRNA expressing models.
Vici syndrome is a severe congenital multisystem disorder characterized by a failure to develop the corpus callosum region of the brain, along with a cardiomyopathy, cataracts, hypopigmentation of the skin, eyes and hair, and a combined immunodeficiency.
To learn more about Taconic's custom model generation, please call 1-888-TACONIC (888-822-6642) in the US or +45 70 23 04 05 in Europe, or email firstname.lastname@example.org.
To learn more about the BeHeard Project, visit http://www.raregenomics.org/beheard-competition/.
About Taconic Biosciences, Inc.
Taconic Biosciences is a fully-licensed, global leader in genetically engineered rodent models and services. Founded in 1952, Taconic helps biotechnology companies and institutions acquire, custom generate, breed, precondition, test, and distribute valuable research models worldwide. Specialists in genetically engineered mouse and rat models, precision research mouse models, and integrated model design and breeding services, Taconic operates three service laboratories and six breeding facilities in the U.S. and Europe, maintains distributor relationships in Asia and has global shipping capabilities to provide animal models almost anywhere in the world.