Genomic surgery by genome editing of pathogenic mutant genes in oral maxillofacial disease-specific iPS cells

2017 Fiscal Year Final Research Report

• Project/Area Number: 16K15823
• Research Category: Grant-in-Aid for Challenging Exploratory Research
• Allocation Type: Multi-year Fund
• Research Field: Surgical dentistry
• Research Institution: Hiroshima University
• Principal Investigator: Okamoto Tetsuji 広島大学, 医歯薬保健学研究科(歯), 教授 (00169153)
• Co-Investigator(Kenkyū-buntansha): 濱田 充子 広島大学, 病院(歯), 歯科診療医 (30760318)
• Project Period (FY): 2016-04-01 – 2018-03-31
• Keywords: 遺伝性顎顔面口腔疾患 / 疾患特異的iPS細胞 / 病原変異遺伝子 / 人工ヌクレアーゼ / ゲノム手術 / 無血清培養 / オーガノイド / von Recallinghausen病

Outline of Final Research Achievements

iPSCs were established from peripheral lymphocytes of healthy subjects and patients with various inherited oral-maxillofacial diseases using serum-free medium hESF 9 and SeVdp, and their cellular characteristics and ability to differentiate into specific cell lineages were examined in serum-free organoid culture. In addition, we compare the differentiation potential to 3 germ layers and the structure of each induction tissue in iPSC- derived teratoma in the dorsal back of SCID mouse. We conducted a full genome analysis of DNA from patients with genetic diseases by the next generation sequencing (NGS), clarified pathogenic mutant genes and other mutations, and aimed to develop a new treatment method for the genetic disease.

Free Research Field

外科系歯学