Technology development for generation in vivo of organ using iPS cells after repair of the disease genome by TALEN

2014 Fiscal Year Final Research Report

• Project/Area Number: 25640106
• Research Category: Grant-in-Aid for Challenging Exploratory Research
• Allocation Type: Multi-year Fund
• Research Field: Medical genome science
• Research Institution: The University of Tokushima
• Principal Investigator: ISHIMARU Yoshiyasu 徳島大学, ソシオテクノサイエンス研究部, 学術研究員 (50435525)
• Project Period (FY): 2013-04-01 – 2015-03-31
• Keywords: ゲノム編集 / CRISPR/Casシステム / TALEN / iPS細胞 / 胚盤胞補完法 / FGF10 / 臓器・器官再生 / ノックイン

Outline of Final Research Achievements

Recently, gene knockout or knock-in technologies that disrupts genome sequence, the transcription activator-like effector nuclease (TALEN) system or the clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated protein (Cas) system, have been developed and used to edit the genomes of various organisms. Our results demonstrate that CRISPR/Cas system more effectively elicits single-step biallelic mutations in mice. In addition, we hypothesized that targeted organs in deficient mice can be produced using a blastocyst complementation in a xenogenic environment. We therefore demonstrate that injection of mouse iPS cells into blastocysts derived from a mutant mouse strain in which the gene necessary to form a particular organ is deficient resulted in generation in vivo of organ almost entirely derived from injected mouse iPS cells.

Free Research Field

再生生物学