A development of novel vectors and an establishment of a new generation of hepatic gene therapy for congenital metabolic diseases

2001 Fiscal Year Final Research Report Summary

• Project/Area Number: 12670798
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Single-year Grants
• Section: 一般
• Research Field: Pediatrics
• Research Institution: Gifu University
• Principal Investigator: KOSAI Ken-ichiro Gifu University School of Medicine ; Department of Cardiovascular Regeneration Science ; Associate Professor, 医学部, 助教授 (90258418)
• Co-Investigator(Kenkyū-buntansha): YOSHINO Makoto Kurume University School of Medicine ; Department of Nutrition and Pediatrics ; Professor, 医学部, 教授 (40080569)
• Project Period (FY): 2000 – 2001
• Keywords: Gene therapy / Congenital metabolic diseases / Adenovirus / Retrovirus / Liver / Chimeric virus / Gutted adenovirus / Gene transduction

Research Abstract

<Aim>
Gene therapy may be the most promising therapeutics for many of congenital metabolic diseases. The largest obstacle of its clinical application is currently no available vector that is capable of in vivo high gene transduction efficiency and the sequential long-term gene expression, both of which are necessary for treating congenital metabolic diseases. The aim of the present study is to develop adeno-retro-chimeric vector and less immunogenic gutted adenoviral vector and to establish novel hepatic gene therapy using them.
<Results>
1. We developed adeno-retro-chimeric vector and studied their function.
2. We developed gutted adenoviral vector and studied their function.
3. We studied mechanisms of hepatic regeneration and hepatocyte death, especially biological roles and therapeutic potentials of hepatocyte growth fadctor and heparin-binding EGF.
4. We developed some novel gene therapy for inveterate diseases using adenoviral vector.
<Future plan>
We will further study the therapeutic potential and adverse effects of these vectors and hepatic gene therapy in various animal models.

Research Products

• [Publications] Shouda T, Yoshida T, Hanada T, Wakioka T, Oishi M, Miyoshi K, Komiya S, Kosai K, Hanakawa Y, Hashimoto K, Nagata K, Yoshimura A.: "Induction of the cytokine signal regulator, SOCS3/CIS3 as a therapeutic strategy for treating inflammatory arthritis"The Journal of Clinical Investigation. 108. 1781-1788 (2001)
  • Description: 「研究成果報告書概要(和文)」より
• [Publications] Shouda T, Yoshida T, Hanada T, Wakioka T, Oishi M, Miyoshi K, Komiya S, Kosai K, Hanakawa Y, Hashimoto K, Nagata K, Yoshimura A.: "Induction of the cytokine signal regulator, SOCS3/CIS3 as a therapeutic strategy for treating inflammatory arthritis"J Clin Invest. 108. 1781-1788 (2001)
  • Description: 「研究成果報告書概要(欧文)」より
• [Publications] Miyado, K., Yamada, G., Yamada, S., Hasuwa, H., Nakamura, Y., Ryu, F., Suzuki, K., Kosai. K., Inoue, K., Ogura, A., Okabe, M., Mekada E.: "CD9 on egg plasma membrane is required for fertilization"Science. 287. 321-324 (2000)
  • Description: 「研究成果報告書概要(欧文)」より