Development of non-viral vector for gene therapy strategy using iPS cells

2011 Fiscal Year Final Research Report

• Project/Area Number: 22890088
• Research Category: Grant-in-Aid for Research Activity Start-up
• Allocation Type: Single-year Grants
• Research Field: Human genetics
• Research Institution: Kyoto University
• Principal Investigator: HOTTA Akitsu 京都大学, iPS細胞研究所, 特定拠点助教 (50578002)
• Research Collaborator: MATSUI Hideto 奈良県立医科大学, 小児科, 講師
• Project Period (FY): 2010 – 2011
• Keywords: 遺伝子治療 / iPS細胞 / 遺伝子導入ベクター / 血友病A

Research Abstract

Aiming for developing a novel gene therapy of hemophilia A, we have developed non-viralbased gene delivery vectors that can maintain foreign gene expression for long term. We identified that new transposon vectors were able to maintain reporter gene expression in human iPS cells for up to 150 days. In addition, we report here for the first time that we can deliver the full-length Factor VIII cDNA with 7. 0 kb in size by using our transposon vectors. Our transposon vector is a promising technology for future gene therapy of severe hemophilia A.

Research Products

• [Journal Article] iPS細胞の小分子制御 (2011)
  • Author(s): 堀田秋津
  • Journal Title: 学術の動向 Volume: Vol.16 Pages: 62-65
• [Presentation] DERIVATION OF NANOG KNOCK-IN GFP REPORTER IPS CELL LINES USING MEGANUCLEASE-INDUCED HOMOLOGOUS RECOMBINATION (2012)
  • Author(s): Duhamel. M, Perillous. S, Epinat. JC, Cedrone. F, Englund. M, Ellerstrom. C, Hyllner. J, Hotta. A, Yamanaka. S, Balbirnie. E, Sourdive. D, Rochon. C
  • Organizer: ISSCR annual meeting
  • Place of Presentation: パシフィコ横浜
  • Year and Date: 2012-06-14
• [Presentation] GENERATION FUNCTIONAL MYOCYTES FROM HUMAN INDUCED PLURIPOTENT STEM CELLS (2012)
  • Author(s): Tanaka. A, Woltjen. K, Hotta. A, Ikeya. M, Shoji. E, Isobe. K, Sakurai. H
  • Organizer: ISSCR annual meeting
  • Place of Presentation: パシフィコ横浜
  • Year and Date: 2012-06-14
• [Presentation] EFFICIENT AND STABLE EXPRESSION OF NON-VIRAL VECTORS IN HUMAN IPS CELLS : TOWARDS A GENE TH ERAPY APPROACH FOR HEMOPHILIA (2012)
  • Author(s): Hotta. A, Fujimoto. N, Nakano.T, Sasakawa. N, Woltjen. K, Gervier. SR, Matsui. H, Yamanaka. S
  • Organizer: ISSCR annual meeting
  • Place of Presentation: パシフィコ横浜
  • Year and Date: 2012-06-13
• [Presentation] SITE SPECIFIC GENOME EDITING OF HUMAN INDUCED PLURIPOTENT STEM CELLS BY PNA (2012)
  • Author(s): Li. H, Fujimoto. N, Sasakawa. N, Shigi. N, Komiyama. M, Hotta. A, Yamanaka. S
  • Organizer: ISSCR annual meeting
  • Place of Presentation: パシフィコ横浜
  • Year and Date: 2012-06-13
• [Presentation] GENERATION OF CANINE IPS CELLS AND THEIR CHARACTERISTIC (2012)
  • Author(s): Hayakawa. K, Kato. T, Akira. N, Ikeya. M, Otsuka. T, Hotta. A, Toguchida. J
  • Organizer: ISSCR annual meeting
  • Place of Presentation: パシフィコ横浜
  • Year and Date: 2012-06-13
• [Presentation] Efficient and stable expression of non-viral vectors in human iPS cells : towards a gene therapy approach for hemophilia (2010)
  • Author(s): Hotta. A
  • Organizer: Canada Stem Cell Network Annua Scientific Meeting
  • Place of Presentation: Calgary, Canada
  • Year and Date: 2010-11-22
• [Remarks]
  • URL: http://www.cira.kyoto-u.ac.jp/hotta/