In Vivo Application of an RNAi Strategy for the Selective Suppression of a Mutant Allele

• Project/Area Number: 20790610
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Single-year Grants
• Research Field: Neurology
• Research Institution: Tokyo Medical and Dental University
• Principal Investigator: KUBODERA Takayuki Tokyo Medical and Dental University, 医学部・附属病院, 医員 (40376801)
• Project Period (FY): 2008 – 2009
• Project Status: Completed (Fiscal Year 2009)
• Budget Amount: ¥4,160,000 (Direct Cost: ¥3,200,000、Indirect Cost: ¥960,000); Fiscal Year 2009: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000); Fiscal Year 2008: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
• Keywords: siRNA / 遺伝子治療 / トランスジェニックマウス / ALS / SOD1 / AAV / AIS

Research Abstract

We previously proposed a strategy for selective suppression of mutant allele. We succeeded in mutant gene suppression in the target organ of mice, using in vivo administered adeno-associated viral vectors. Our method may prove useful for siRNA-based gene therapy for inherited diseases.

Research Products

• [Journal Article] Silencing efficiency differs among tissues and endogeneous microRNA pathway is preserved in short hairpin RNA transgenic mice (2009)
  • Author(s): sasaguri H, Mitani T, Anzai M, Kubodera T, Saito Y, Yamada H, Mizusawa H, Yokota T
  • Journal Title: FEBS Letters 583 Pages: 213-218
  • Peer Reviewed
• [Journal Article] Silencing efficiency differs among tissues and endogeneous microRNA pathway is preserved in short hairpin RNA transgenic mice (2009)
  • Author(s): Sasaguri H, Mitani T, Anzai M, Kubodera T, Saito Y, Yamada H, Mizusawa H, Yokota T
  • Journal Title: FEBS Letters 583 Pages: 213-218
  • Peer Reviewed
• [Journal Article] ウイルスベクターを用いたshRNAによる遺伝子治療の現状と問題点 (2008)
  • Author(s): 久保寺隆行, 横田隆徳
  • Journal Title: Antisense 12 Pages: 37-47
• [Journal Article] ALSの遺伝子治療 (2008)
  • Author(s): 久保寺隆行, 横田隆徳
  • Journal Title: Clinical Neuroscience Pages: 337-339
• [Journal Article] ALSの遺伝子治療 (2008)
  • Author(s): 久保寺隆行, 横田隆徳
  • Journal Title: Clinical Neuroscience 26 Pages: 337-339
• [Presentation] siRNA発現アデノ随伴ウイルスベクターを用いた家族性アミロイドポリニューロパチー(FAP)の遺伝子治療 (2009)
  • Author(s): 久保寺隆行, 大平進嘉, 横田隆徳, 水澤英洋
  • Organizer: 第50回日本神経学会総会
  • Place of Presentation: 仙台国際センター
• [Presentation] shRNA-Mediated Gene Silencing in Non-Human Primates with AAV8 (2009)
  • Author(s): Takayuki Kubodera, Shinga Ohira, Yuko Katakai, Hirofumi Akari, Yukihiko Hirai, Takashi Shimada, Hiroaki Mizukami, Keiya Ozawa, Hidehiro Mizusawa, Takanori Yokota
  • Organizer: 第15回日本遺伝子治療学会学術集会
  • Place of Presentation: 大阪大学コンベンションセンター
• [Presentation] In vivo Application of a New RNAi Strategy for selective Suppression of a Mutant Allele to Any Mutation. (2008)
  • Author(s): Kubodera T, Yamada H, Ito K, Anzai M, Mitani T, Mizusawa H, Yokota T
  • Organizer: ASGT 11th Annual Meeting
  • Place of Presentation: Boston, Massachusettes
• [Presentation] 変異遺伝子特異的なRNA干渉法のin vivoへの応用 (2008)
  • Author(s): 久保寺隆行, 山田宏美, 横田隆徳, 水澤英洋
  • Organizer: 第49回日本神経学会総会
  • Place of Presentation: 横浜
• [Presentation] In vivo Application of a New RNAi Strategy for selective Suppression of a Mutant Allele to Any Mutation. (2008)
  • Author(s): Kubodera T, Yamada H, Ito K, Anzai M, Mitani T, Miiusawa H, Yokota T
  • Organizer: ASGT 11^<th> Annual Meeting
  • Place of Presentation: Boston, Massachusettes
• [Remarks]
  • URL: http://www.tmd.ac.jp/med/nuro/study.html#b
• [Remarks]
  • URL: http://www.tmd.ac.jp/med/nuro/study.html#b
• [Remarks]
  • URL: http://www.tmd.ac.jp/med/nuro/study.html#b