Lentiviral vector mediated gene therapy for Krabbe disease

• Project/Area Number: 20591233
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Single-year Grants
• Section: 一般
• Research Field: Pediatrics
• Research Institution: Jikei University School of Medicine
• Principal Investigator: KOBAYASHI Hiroshi Jikei University School of Medicine, 医学部, 講師 (90266619)
• Co-Investigator(Kenkyū-buntansha): KIMURA Takahiro 東京慈恵会医科大学, 医学部, 助教 (00307430) ; KAWAI Toshinao 東京慈恵会医科大学, 医学部, 助教 (20328305)
• Project Period (FY): 2008 – 2010
• Project Status: Completed (Fiscal Year 2010)
• Budget Amount: ¥4,680,000 (Direct Cost: ¥3,600,000、Indirect Cost: ¥1,080,000); Fiscal Year 2010: ¥650,000 (Direct Cost: ¥500,000、Indirect Cost: ¥150,000); Fiscal Year 2009: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000); Fiscal Year 2008: ¥2,210,000 (Direct Cost: ¥1,700,000、Indirect Cost: ¥510,000)
• Keywords: 遺伝 / 先天異常学 / 遺伝子治療 / Krabbe病 / レンチウイルスベクター / レンチウイルス / クラッベ病 / GALC / GFP

Research Abstract

We investigated the effect of lenti-virus mediated gene therapy for Krabbe disease. We succeeded in preparing high titer lenti-virus including GALC, deficient enzyme in Krabbe disease expressing gene by large scale harvesting and ultracentrifuge, and injected for neonatal twitcher mouse, model for Krabbe disease resulted in efficient improvement in nutrition and life span. And we had more efficient improvement in the phenotype of twitcher mice by the combination of gene therapy and substrate reduction therapy using L-cycloserine.

Research Products

• [Presentation] (2010)
  • Author(s): Kobayashi H., Ariga M., et al.
  • Organizer: 16^<th> Japanese Society of Gen and Cell Therapy.
  • Place of Presentation: Tochigi., Japan
  • Year and Date: 2010-07-01
• [Presentation] Neonatal Gene Therapy for the mouse model of Krabbe Disease. (2010)
  • Author(s): Kobayashi_H., Ariga M., et al.
  • Organizer: 16^<th> Japanese Society of Gene and Cell Therapy
  • Place of Presentation: Tochigi, Japan
  • Year and Date: 2010-07-01
• [Presentation] (2010)
  • Author(s): 小林博司、有賀賢典, ほか
  • Organizer: 第113回日本小児科学会
  • Place of Presentation: 盛岡
  • Year and Date: 2010-04-24
• [Presentation] レンチウイルスベクターシステムを用いたクラッベ病の遺伝子治療 (2010)
  • Author(s): 小林博司, ほか
  • Organizer: 第113回日本小児科学会
  • Place of Presentation: 盛岡
  • Year and Date: 2010-04-24
• [Remarks]
  • URL: http://jikei-dna.jp/
• [Remarks]
  • URL: http://www.nanbyou.or.jp/sikkan/117.htm