Gene therapy of Hepatitis C using monkey by siRNA

• Project/Area Number: 18390215
• Research Category: Grant-in-Aid for Scientific Research (B)
• Allocation Type: Single-year Grants
• Section: 一般
• Research Field: Gastroenterology
• Research Institution: Tokyo Medical and Dental University
• Principal Investigator: YOKOTA Takanori Tokyo Medical and Dental University, Department of Neurology and Neurological Science, Associate Professor (90231688)
• Co-Investigator(Kenkyū-buntansha): AKARI Hirofumi National Institute of Biomedical Innovation, Tsukuba Primate Research Center, 研究リーダー (20294671) ; ENOMOTO Nobuyuki Yamanashi University, First Department of Medicine, 教授 (20251530)
• Project Period (FY): 2006 – 2007
• Project Status: Completed (Fiscal Year 2007)
• Budget Amount: ¥17,750,000 (Direct Cost: ¥15,500,000、Indirect Cost: ¥2,250,000); Fiscal Year 2007: ¥9,750,000 (Direct Cost: ¥7,500,000、Indirect Cost: ¥2,250,000); Fiscal Year 2006: ¥8,000,000 (Direct Cost: ¥8,000,000)
• Keywords: RNA interference / siRNA / AAV / Tamarin / shRNA / marmoset / GBV-B / Vitamin E / 新世界ザル / カチオニックベクター / C型肝炎 / AAV8 / インターフェロン

Research Abstract

1) We succeeded in preventing induction of interferon after intravenous administration of siRNA/cationic liposome by chemical modifications including 2'O-methylation, phosphorothionate to siRNA oligonucleotides, resulting in increase the dose up to 20mg/kg. With this new siRNA with chemical modifications, GBV-B inoculated tamarins were treated. However, viral titres were not decreased, although transaminases tended to be decreased.
2)As a new siRNA vector, we use the vitamin E as a carrier molecule of siRNA in vivo which has -a physiological transport pathway to most of organs. The a-tocopherol was covalently bound to anti-sense strand of 27/29 mer siRNA at the 5' end (Toc-siRNA). The 27/29 mer Toc-siRNA was designed to be cleaved by Dicer in the cytosol and to produce 21/21 mer maturated siRNA after releasing a-tocopherol. The anti-oxidant activity of a-tocopherol in Toc-siRNA was abolished. With this new delivery strategy, systemic and intravenous injection of clinically relevant dose (2 mg/kg) of Toc-siRNA targeting apolipoprotein B(apoB) can reduce endogenous apoB mRNA in the liver by about 80%. The down-regulation of apoB was confirmed by the observation of lipid droplets accumulation in the liver as a phenotype. Neither induction of interferons nor other overt side effects were observed in biochemical and pathological analysis. These findings demonstrate the therapeutic potential of a-tocopherol-bound siRNAs for the treatment of disease.

Research Products

• [Journal Article] Efficient In Vivo Delivery of siRNA to the Liver by Conjugation of α-Tocopherol. (2008)
  • Author(s): Nishina K, Unno T, Uno Y, Kubodera T, Kanouchi T, Mizusawa H, Yokota T:
  • Journal Title: Molecular Therapy 16 Pages: 734-740
  • Description: 「研究成果報告書概要(和文)」より
  • Peer Reviewed
• [Journal Article] Inhibition of hepatitis C virus infection and expression in vivo by recombinant adenovirus expressing short hairpin RNA. (2008)
  • Author(s): Sakamoto N, Tanabe Y, Yokota T, Saito K, Sekine-Osajima Y, Nakagawa M, Itsui Y, Tasaka M, Sakurai Y, Chen CH, Yano M, Ohkoshi S, Aoyagi Y, Maekawa S, Enomoto N, Kohara M, Watanabe M
  • Journal Title: J Gastro Hepatol (印刷中)
  • Description: 「研究成果報告書概要(和文)」より
  • Peer Reviewed
• [Journal Article] Efficient in vivo delivery of siRNA to liver by conjugation of a-tocopherol (2008)
  • Author(s): Nishina K. Unno T ; Uno Y. Kubodera T,. Kanouchi T, Mizusawa H, Yokota T
  • Journal Title: Mol Ther 16 Pages: 734-740
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] Efficient In Vivo Delivery of siRNA to the Liver by Conjugation of a -Tocopherol (2008)
  • Author(s): Kazutaka Nishina, Toshinori Unno, Yoshitaka Uno, Takayuki Kubodera, Tadashi Kanouchi, Hidehiro Mizusawa Takanori Yokota
  • Journal Title: Molecular Therapy 16 Pages: 734-740
  • Peer Reviewed
• [Journal Article] Inhibition of hepatitis C virus infection and expression in vitro and in vivo by recombinant adenovirus expressing short hairpin RNA (2008)
  • Author(s): Sakamoto N, Tanabe Y, Yokota T, Saito K, Sekine-Osajima Y, Nakagawa M, Itsui Y, Tasaka M, Sakurai Y, Chen CH, Yano M, Ohkoshi S, Aoyagi Y, Maekawa S, Enomoto N, Kohara M, Watanabe M
  • Journal Title: J Gastro Hepatol (印刷中)
  • Peer Reviewed
• [Journal Article] Efficient regulation of viral replication by siRNA in a non - human primate surrogate model for hepatitis C. (2007)
  • Author(s): Ybkota T, Iijima S, Kubodera T, Ishii K, Katakai Y, Ageyama N, Chen YW, Lee YJ, Unno T, Nishina K, Iwasaki Y, Maki N, Mizusawa H, Akari H
  • Journal Title: Biochem Biophys Res Com 361 Pages: 294-300
  • Description: 「研究成果報告書概要(和文)」より
  • Peer Reviewed
• [Journal Article] Increase of disease duration of amyotrophic lateral sclerosis in a mouse model bytransgenic small interfering RNA. (2007)
  • Author(s): Yokota T, Sasaguri H, Saito Y, Yamada H, Unno N, Yamamoto T, Kubodera T, Anzai M, Mitani T, Mizusawa H.
  • Journal Title: Archives Neurology 64 Pages: 145-146
  • Description: 「研究成果報告書概要(和文)」より
  • Peer Reviewed
• [Journal Article] Bax-inhibiting peptide protects cells from polyglutamine toxicity caused by Ku70 acetylation. (2007)
  • Author(s): Li Y, Yokota T, Gama V, Yoshida T, Gomez JA, Ishikawa K, Sasaguri H, Cohen HY, Sinclair D A, Mizusawa H, Matsuyama S
  • Journal Title: Cell Death Differ 14 Pages: 2058-2067
  • Description: 「研究成果報告書概要(和文)」より
  • Peer Reviewed
• [Journal Article] GBV-B as a pleiotropic virus: distribution of GBV-B in extrahepatic tissues in vivo. (2007)
  • Author(s): Ishii K, Iijima S, Kimura N, Lee YJ, Ageyama N Yagi S, Yamaguchi K, Maki N, Mori K, Yoshizaki S, Machida S, Suzuki T, Iwata N, Sata T, Terao K, Miyamura T, Akari H
  • Journal Title: Microbes Infect. 9 Pages: 515-521
  • Description: 「研究成果報告書概要(和文)」より
  • Peer Reviewed
• [Journal Article] Bax-inhibiting peptide protects cells from polyglutamine toxicity caused by Ku 70 acelylation (2007)
  • Author(s): Li Y. Yokota T, Gama V. Yoshida T, Gomez JA, Ishikawa K, Sasaguri H. Cohen NY Sinclair DA, Mizusawa H. Matsuyama S
  • Journal Title: Cell Death Differ 14 Pages: 2058-2067
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] Efficient regulation of viral replication by siRNA in a non-human primate surrogate model for hepatitis C. (2007)
  • Author(s): Yokota T, lijima S, Kubodera T, Ishii K, Katakai Y, Ageyama N, Chen YW, Lee YJ, Unno T, Nishina K, Iwasaki Y. Maki N, Mizusawa H, Akari H
  • Journal Title: Biochem Biophys Res Com 361 Pages: 294-300
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] Increase of disease duration of amyotrophic lateral sclerosis in a mouse model by transgenic small interfering RNA (2007)
  • Author(s): Yokota T. Sasaguri H, Saito Y. Yamada H, Unno N, Yamamoto T Kubodera T, Anzai M. Mitani T, Mizusawa H
  • Journal Title: Arch Neurol 64 Pages: 145-146
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] GBV-B as a pleiotropic virus : distribution of GBV B in extrahepatic tissues in vivo (2007)
  • Author(s): Ishii K, lijima S, Kimura N, Lee YJ, Ageyama N, Yagi S, Yamaguchi K, Maki N ; Mori K, Yoshizaki S. Machida S, Suzuki T, Iwvata N, Sata T, Terao K. Miyamura T, Akari H.
  • Journal Title: Microbes Infect 9 Pages: 515-521
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] Clinical application of RNAi to neurodegenerative disease "Science for neurodegenerative disease" (2007)
  • Author(s): Yokota T, Takah ashi R.
  • Journal Title: Nanzan-do
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] Ed by Takaku H, Yamamoto N. siRNA-based gene therapy for autosomal dominant disease of the nervous system. In RNAi therapeutics (2007)
  • Author(s): Yokota T.
  • Journal Title: Transworld Research Network
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] Clinical application of siRNA (2007)
  • Author(s): Yokota T.
  • Journal Title: Neuro
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] Gene Therapy of Hereditary Diseases with RNA Interference (2007)
  • Author(s): Yokota T, Kubodera T. Yamada H, Unno T. Nishina K. Mitani T, Mizusawa H.
  • Journal Title: Joint Annual Meeting of the Molecular Biology Society of Japan and the Japanese Biochemical Society
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] Efficient regulation of viral replication by siRNA in a non-human primate surrogate model for hepatitis C. (2007)
  • Author(s): Yokota T, Iijima S, Kubodera T, Ishii K, Katakai Y, Ageyama N, Chen YW, Lee YJ, Unno T, Nishina K, Iwasaki Y, MakiN, Mizusawa H, Akari H
  • Journal Title: Biochem Biophys Res Com 361 Pages: 294-300
  • Peer Reviewed
• [Journal Article] Increase of disease duration of amyotrophic lateral sclerosis in a mouse model by transgenic small interfering RNA (2007)
  • Author(s): Yokota T, Sasaguri H, Saito Y, Yamada H, Unno N, Yamamoto T, Kubodera T, Anzai M, Mitani T, Mizusawa H.
  • Journal Title: Archives Neurology 64 Pages: 145-146
  • Peer Reviewed
• [Journal Article] siRNA干渉によるALSの治療戦略 (2007)
  • Author(s): 横田隆徳
  • Journal Title: Brain and Nerve 59 Pages: 1187-1194
• [Journal Article] Increase of disease duration of amyotrophic lateral sclerosis in a nouse model by transgenic small interfering RNA. (2007)
  • Author(s): Yokoya T, Sasaguri H, Yamada H, Unno N, Yamamoto T, Kubodera T, Anzai M, Mitani T, Mizusawa H
  • Journal Title: Arch Neurol 64 Pages: 145-146
• [Journal Article] siRNAを用いたALSの遺伝子治療 (2007)
  • Author(s): 横田隆徳
  • Journal Title: 医学のあゆみ。 (印刷中)
• [Journal Article] In vivo delivery of small interfering RNA targeting brain capillary endothelial cells. (2006)
  • Author(s): Hino T, Yokota T, Ito S, Nishina K, Kang YS, Mori S, Hori S, Kanda T, Terasaki T, Mizusawa H:
  • Journal Title: Biochem Biophys Res Com 340 Pages: 263-267
  • Description: 「研究成果報告書概要(和文)」より
  • Peer Reviewed
• [Journal Article] 脳血管内皮細胞をターゲットとしたsiRNAを用いた遺伝子治療の可能性 (2006)
  • Author(s): 横田隆徳
  • Journal Title: Neuroimmunology 14 Pages: 141-145
  • Description: 「研究成果報告書概要(和文)」より
• [Journal Article] In vivo delivery of small interfering RNA targeting brain capillary endothelial cells (2006)
  • Author(s): Hino T, Yokota T. Ito S. Nishina K, Kang YS, Mori S, Hori S. Kanda T. Terasald T. Mizusativa H
  • Journal Title: Biochem Biophys Res Commun 340 Pages: 263-267
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] Efficient regulation of viral replication by systemically administered siRNA in a non-human primate surrogate model for hepatitis C. (2006)
  • Author(s): Yokota T. Kubodera T, Unno N, Chen Y, Nishina K. Mizusawa H, Akari H.
  • Journal Title: 12th annual meeting of Japan Society of Gene Therapy
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] Efficient regulation of viral replication by systemically administered siRNA in a non-human primate surrogate model for hepatitis (2006)
  • Author(s): Yokota T, lijima S. Kubodera T. Ishii K. Ageyama N. Yingwei C. Lee J-J, Maki N, Mizusawa H, Akari H.
  • Journal Title: 12th annual meeting of Japan Society of Gene Therapy
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] Adeno-associated virus serotype 8(AAV-8)-mediated shRNA delivery in vivo (2006)
  • Author(s): Chen Y, Yokota T, Kubodera T. Ohishi I, Li Y. Nishina K. Hirai Y. Shimada T. Mizusawa H.
  • Journal Title: 12th annual meeting of Japan Society of Gene Therapy
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] Efficient delivery of siRNA targeting brain endothelial cells (2006)
  • Author(s): Nishina K, Yokota T, Hino T, Yoshida M, Terasaki T, MizusaNva H.
  • Journal Title: 12th annual meeting of Japan Society of Gene Therapy
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] In viva analysis of new RNAi strategy : selective suppression of a mutant allele to any mutation (2006)
  • Author(s): Kubodera T, Yokota T, Saito Y, Mizusawa H.
  • Journal Title: 12th annual meeting of Japan Society of Gene Therapy
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] Tranegenic siRNA holls ALS in a mouse model (2006)
  • Author(s): Yokota T. Sasaguri H, Saito Y. Mitani T. Mizusawa H.
  • Journal Title: Neuro
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] Liver toxicity induced by AAV delivered short hairpin RNA (2006)
  • Author(s): Unno T. Kubodera T. Murayama S, Ohira S. Nishina K. Igari T, Eishi Y. Hirai Y. Shimada T, Yokota T, Mizusawa H.
  • Journal Title: 13th annual meeting of Japan Society of Gene Therapy
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] A new RNAi strategy for selective suppression of a mutant allele to anv mutation (2006)
  • Author(s): Yamada H. Kubodera T, Yokota T. Mizusawa H.
  • Journal Title: 13th annual meeting of Japan Society of Gene Therapy
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] In vivo delivery of small interfering RNA targeting brain capillary endothelial cells. (2006)
  • Author(s): Hino T, Yokoya T, Ito S, Nishina K, Kang YS, Mori S, Kanda T, Terasaki T, Mizusawa H.
  • Journal Title: Biochem Biophys Res Commun. 340 Pages: 263-267
• [Journal Article] siRNAiの核酸医薬としての可能性. (2006)
  • Author(s): 横田隆徳
  • Journal Title: 分子細胞治療 5 Pages: 231-236
• [Journal Article] RNA干渉によるアルツハイマー病治療の可能性 (2006)
  • Author(s): 横田隆徳
  • Journal Title: Progress in Medicine 26 Pages: 441-445
• [Journal Article] Negative regulation of intracellular hepatitis C virus replication by interferon regulatory factor 3. (2006)
  • Author(s): Yamashiro T, Sakamoto N, Kurosaki M, Kanazawa N, Tanabe Y, Nakagawa M, Chen CH, Itsui Y, Koyama T, Takeda Y, Maekawa S, Enomoto N, Sakugawa H, Watanabe M
  • Journal Title: J Gastroenterol. 41 Pages: 750-757
  • NAID: 10018255879
• [Journal Article] Expressional screening of interferon-stimulated genes for antiviral activity against hepatitis C virus replication. (2006)
  • Author(s): Itsui Y, Sakamoto N, Kurosaki M, Kanazawa N, Tanabe Y, Koyama T, Takeda Y, Nakagawa M, Kakinuma S, Sekine Y, Maekawa S, Enomoto N, Watanabe M.
  • Journal Title: J Viral Hepat. 13 Pages: 690-700
• [Journal Article] Site-specific mutation of the interferon sensitivity-determining region (ISDR) modulates hepatitis C virus replication. (2006)
  • Author(s): kohashi T, Maekawa S, Sakamoto N, Kurosaki M, Watanabe H, Tanabe Y, Chen CH, Kanazawa N, Nakagawa M, Kakinuma S, Yamashiro T, Itsui Y, Koyama T, Enomoto N, Watanabe M.
  • Journal Title: J Viral Hepat. 13 Pages: 582-90
• [Journal Article] Analysis of prognostic factors in therapeutic responses to inteerferon in patients with chronic hepatitis C. (2006)
  • Author(s): Hosogaya S, Ozaki Y, Enomoto N, Akahane Y.
  • Journal Title: Transl Res. 148 Pages: 79-86
• [Journal Article] ALS-linked mutant SOD1 induces ER stress- and ASK1-dependent motorneuron death by targeting Derlin-1.
  • Author(s): Nishitoh H, Kadowaki H, Nagai A, Maruyama T, Yokota T, Fukutomi H, Noguchi T, Matsuzawa A, Takeda K, Ichijo H
  • Journal Title: Gene Dev (印刷中)
  • Description: 「研究成果報告書概要(和文)」より
  • Peer Reviewed
• [Journal Article] Inhibition of hepatitis C virus infection and expression in vitro and in vivo by recombinant adenovi us expressing short hairpin RNA
  • Author(s): Sakamoto N. Tanabe Y, Yokota T, Saito K, Sekine-Osajima Y, Nakagawa M, Itsui Y, Tasaka M, Sakurai Y Chen CH, Yano M, Ohkoshi S. Aoyagi Y, Maekawa S, Enomoto N, Kohara M, Watanabe M
  • Journal Title: J Gastro Hepatol (in press)
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] ALS-linked mutant SOD I induces ER stress- and ASK I -dependent motor neuron death by targeting Derlin-1
  • Author(s): Nishitoh H, Kadowaki H, Nagai A. Maruyama T, Yokota T. Fukutomi H, Noguchi T, Matsuyama A, Takeda K, Ichijo H.
  • Journal Title: Gene Dev (in press)
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] Gene therapy with siRNA targeting brain endothelial cells
  • Author(s): Yokota T.
  • Journal Title: Neuroimmunology
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] Efficient regulation of viral replication by siRNA in a non-human primate surrogate model for hepatitis C.
  • Author(s): Yokota T. Kubodera T, Hirabayashi K, Ishii K. Maki N, Mizusawa H, En.omoto N. Yano J, Akari H.
  • Journal Title: 20th The Japan Society of Hepatology
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] In vivo application of siRNA/shRNA
  • Author(s): Yokota T.
  • Journal Title: Bio-Japan Symposium
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] Tranegenic siRNA holts ALS in a mouse model
  • Author(s): Yokota T. Sasaguri H. Saito Y, Mitani T, Mizusawa H.
  • Journal Title: 59th AAN annual meeting
  • Description: 「研究成果報告書概要(欧文)」より
• [Journal Article] Efficient in vivo delivery of siRNA to liver by conjugation of a-tocopherol
  • Author(s): Yokota T, Unno T, Nishina K, Uno Y. Kubodera T, Kanouchi T, Mizusawa H.
  • Journal Title: Keystone Symposim, RNAi, miRNA and non-coding RNA
  • Description: 「研究成果報告書概要(欧文)」より
• [Presentation] Efficient in vivo delivery of siRNA to liver by conjugation of a-toco pherol. (2008)
  • Author(s): Yokota T, Unno T, Nishina K, Uno Y, Kubodera T, Kanouchi T, Mizusawa H:
  • Organizer: Keystone Symposim, RNAi, miRNA and non-coding RNA
  • Place of Presentation: Whistler, Canada
  • Year and Date: 2008-03-28
  • Description: 「研究成果報告書概要(和文)」より
• [Presentation] Gene Therapy of Hereditary Diseases with RNA Interference. (2007)
  • Author(s): 横田隆徳, 久保寺隆行, 山田宏美, 海野敏紀, 仁科一隆, 三谷 匡, 水澤英洋.
  • Organizer: 第30回日本分子生物学会年会, 第80回日本生化学学会大会
  • Place of Presentation: 横浜
  • Year and Date: 2007-12-13
  • Description: 「研究成果報告書概要(和文)」より
• [Presentation] Clinical application of siRNA. (2007)
  • Author(s): Yokota T
  • Organizer: Neuro2007
  • Place of Presentation: Yokohama
  • Year and Date: 2007-09-04
  • Description: 「研究成果報告書概要(和文)」より
• [Presentation] Liver toxicity induced by AAV-delivered short hairpin RNA. (2007)
  • Author(s): Unno T, Kubodera T, Murayama S, Ohira S, Nishina K, Igari T, Eishi Y, Hirai Y, Shimada T, Yokota T, Mizusawa H
  • Organizer: 第13回日本遺伝子治療学会
  • Place of Presentation: 東京
  • Year and Date: 2007-06-30
  • Description: 「研究成果報告書概要(和文)」より
• [Presentation] A new RNAi strategy for selective suppression of a mutant allele to any mutation. (2007)
  • Author(s): Yamada H, Kubodera T, Yokota T, Mizusawa H.
  • Organizer: 第13回日本遺伝子治療学会
  • Place of Presentation: 東京
  • Year and Date: 2007-06-30
  • Description: 「研究成果報告書概要(和文)」より
• [Presentation] Tranegenic siRNA holts ALS in a mouse model. (2007)
  • Author(s): Yokota T, Sasaguri H, Saito Y, Mitani T, Mizusawa H
  • Organizer: 59th AAN annual meeting
  • Place of Presentation: Boston
  • Year and Date: 2007-05-04
  • Description: 「研究成果報告書概要(和文)」より
• [Presentation] Tranegenic siRNA holts ALS in a mouse model. (2006)
  • Author(s): Yokota T, Sasaguri H, Saito Y, Mitani T, Mizusawa H
  • Organizer: Neuro2006
  • Place of Presentation: Atanta
  • Year and Date: 2006-10-16
  • Description: 「研究成果報告書概要(和文)」より
• [Presentation] In vivo application of siRNA/shRNA. (2006)
  • Author(s): Yokota T
  • Organizer: Bio-Japan Symposium
  • Place of Presentation: Osaka, Japan
  • Year and Date: 2006-09-15
  • Description: 「研究成果報告書概要(和文)」より
• [Presentation] Efficient regulation of viral replication by systemically administered siRNA in a non-human primate surrogate model for hepatitis C. (2006)
  • Author(s): Yokota T, Kubodera T, Unno N, Chen Y, Nishina K, Mizusawa H, Akari H
  • Organizer: 12th annual meeting of Japan Society of Gene Therapy 2006
  • Place of Presentation: Tokyo
  • Year and Date: 2006-08-26
  • Description: 「研究成果報告書概要(和文)」より
• [Presentation] Adeno-associated virus serotype 8(AAV-8)mediated shRNA delivery in vivo. (2006)
  • Author(s): Chen Y, Yokota T, Kubodera T, Ohnishi I, Li Y, Nishina K, Hirai Y, Shimada T, Mizusa wa H.
  • Organizer: 第12回日本遺伝子治療学会
  • Place of Presentation: 東京
  • Year and Date: 2006-08-25
  • Description: 「研究成果報告書概要(和文)」より
• [Presentation] Efficient delivery of siRNA targeting brain endothelial cells. (2006)
  • Author(s): Nishina K, Yokota T, Hino T, Yoshida M, Terasaki T, Mizusawa H
  • Organizer: 第12回日本遺伝子治療学会
  • Place of Presentation: 東京
  • Year and Date: 2006-08-25
  • Description: 「研究成果報告書概要(和文)」より
• [Presentation] siRNAの静脈注射によるC型肝炎の遺伝子治療:サルを用いたC型肝炎サロゲートモデルによる検討 (2006)
  • Author(s): 横田隆徳, 久保寺隆行, 平林加寿子, 石井孝司, 愼 昇, 水澤英洋, 榎本信幸, 矢野純一, 明里宏文
  • Organizer: 第20回日本肝臓学会
  • Place of Presentation: 京都
  • Year and Date: 2006-05-26
  • Description: 「研究成果報告書概要(和文)」より
• [Presentation] Efficient regulation of viral replication by systemically administered siRNA in a non-human primate surrogate model for hepatitis. (2006)
  • Author(s): Yokota T, Iijima S, Kubodera T, Ishii K, Ageyama N, Yingwei C, Lee J-J, Maki N, Mizusawa H, Akari H
  • Organizer: 第12回日本遺伝子治療学会
  • Place of Presentation: 東京
  • Description: 「研究成果報告書概要(和文)」より
• [Presentation] In vivo analysis of new RNAi strategy; selective suppression of a mutant allele to any mutation. (2006)
  • Author(s): Kubodera T, Yokota T, Saito Y, Mizusawa H
  • Organizer: 第12回日本遺伝子治療学会
  • Place of Presentation: 東京
  • Description: 「研究成果報告書概要(和文)」より
• [Book] RNAiを用いた神経変性疾愚への臨床応用。「神経変性疾患のサイエンス」 (2007)
  • Author(s): 横田隆徳:高橋良輔編
  • Total Pages: 10
  • Publisher: 南山堂
  • Description: 「研究成果報告書概要(和文)」より
• [Book] siRNA-based gene therapy for autosomal dominant disease of the nervous system. In RNAi therapeutics. (2007)
  • Author(s): Yokota T. Ed by Takaku H, Yamamoto N.
  • Total Pages: 5
  • Publisher: Transworld Research Network
  • Description: 「研究成果報告書概要(和文)」より
• [Patent(Industrial Property Rights)] Gene delivery of siRNA with new endogenous lipoprotein. (2007)
  • Inventor(s): Yokota T, Nishina K, Mizusawa H Unno T
  • Industrial Property Rights Holder: 東京医科歯科大学
  • Filing Date: 2007-11-30
  • Description: 「研究成果報告書概要(和文)」より
  • Overseas
• [Patent(Industrial Property Rights)] Gene delivery of siRNA with new endogenous lipoprotein. (2007)
  • Inventor(s): Yokota T, Nishina K, Mizusawa H Unno T,
  • Industrial Property Rights Holder: 東京医科歯科大学
  • Filing Date: 2007-11-30
  • Overseas