Basic studies toward CRISPR/Cas9-mediated cell therapy

2016 Fiscal Year Final Research Report

• Project/Area Number: 26461530
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Research Field: Pediatrics
• Research Institution: Kitasato University
• Principal Investigator: Miyashita Toshiyuki 北里大学, 医学部, 教授 (60174182)
• Co-Investigator(Renkei-kenkyūsha): NAGAO Kazuaki 北里大学, 医学部, 講師 (60392487) ; KAMEYAMA Kohzoh 北里大学, 医学部, 講師 (40214556) ; TAKAYAMA Yoshinaga 北里大学, 医学部, 講師 (90245407)
• Project Period (FY): 2014-04-01 – 2017-03-31
• Keywords: 母斑基底細胞癌症候群 / CRISPR/Cas9システム / iPS細胞 / 遺伝子編集

Outline of Final Research Achievements

We have successfully established nevoid basal cell carcinoma syndrome (NBCCS)-specific induced pluripotent stem cells (iPS cells). Medulloblastoma was formed in all the teratomas generated from all NBCCS-specific iPSC clones established from 4 different patients. These results demonstrated that NBCCS-specific iPSCs can be a good model of NBCCS-derived medulloblastoma, and thereby used for drug screening to prevent or treat medulloblastomas.
In addition, by using CRISPR/Cas9 system, we also generated iPS cells in which mutation was introduced in remaining normal PTCH1 allele. These cells exhibited accelerated cell growth and expression of Gli target genes indicating up-regulation of hedgehog signaling. Moreover, we generated iPS cells in which mutated allele was edited and corrected.

Free Research Field

分子遺伝学