Presentation Information
[O23-3]Development of a Gene Panel and SSO Therapy Targeting Deep Intronic Variants in Citrin Deficiency
Eri Imagawa1, Jin Rong Ow2, Feng Chen3, Wei Yuan Cher2, Shermin Yu Tung Chan2, Rajasekhar Reddy Gurrampati2, Venkataramanan Ramadass2, Mun Fai Loke4, Tommaso Tabaglio2, Yoko Manome5, Hirotaka James Okano5, Masahide Yazaki6, Gaik Siew Ch’ng7, Manikandan Lakshmanan2, Su Seong Lee8, Jackie Y. Ying9, Ernesto Guccione110, Kimihiko Oishi1, Keng Boon Wee2 (1.Department of Pediatrics, The Jikei University School of Medicine, Tokyo, Japan, 2.Institute of Molecular and Cell Biology (IMCB), Agency for Science, Technology and Research (A*STAR), Singapore, 3.King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia, 4.Camtech Biomedical Pte Ltd, Singapore, 5.Division of Regenerative Medicine, The Jikei University School of Medicine, Tokyo, Japan, 6.Institute for Biomedical Sciences, Shinshu University, Matsumoto, Japan, 7.Department of Genetics, Penang General Hospital, Penang, Malaysia, 8.Department of Bioengineering and Nanomedicine, King Faisal Specialist Hospital & Research Centre, Riyadh, Saudi Arabia, 9.Department of Bioengineering, King Fahd University of Petroleum & Minerals, Dhahran, Saudi Arabia, 10.Center for OncoGenomics and Innovative Therapeutics (COGIT), Center for Therapeutics Discovery, Department of Oncological Sciences and Pharmacological Sciences, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, USA)
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