Screening methods to discover ATTR amyloid disrupters

• Project/Area Number: 18K07502
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Review Section: Basic Section 52020:Neurology-related
• Research Institution: Kumamoto University
• Principal Investigator: Ueda Mitsuharu 熊本大学, 大学院生命科学研究部(医), 教授 (60452885)
• Co-Investigator(Kenkyū-buntansha): 水口 峰之 富山大学, 学術研究部薬学・和漢系, 教授 (30332662) ; 山中 邦俊 熊本大学, 発生医学研究所, 准教授 (90212290)
• Project Period (FY): 2018-04-01 – 2021-03-31
• Project Status: Completed (Fiscal Year 2020)
• Budget Amount: ¥4,550,000 (Direct Cost: ¥3,500,000、Indirect Cost: ¥1,050,000); Fiscal Year 2020: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000); Fiscal Year 2019: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000); Fiscal Year 2018: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
• Keywords: アミロイドブレイカー / アミロイドーシス / トランスサイレチン / 薬剤スクリーニング / Drug repositioning / アミロイド・ブレイカー

Outline of Final Research Achievements

Using the highly amyloidogenic TTR81-127 fragment, we developed a cell-based high-throughput screening method to discover compounds that disrupt TTR amyloid fibrils. Screening a library of 1280 off-patent drugs, we identified two candidate repositioning drugs, pyrvinium pamoate and apomorphine hydrochloride.
Both drugs disrupted patient-derived TTR amyloid fibrils ex vivo, and pyrvinium pamoate also stabilized the tetrameric structure of TTR ex vivo in patient plasma. We conclude that our TTR81-127 based screening method is very useful for discovering therapeutic drugs that directly disrupt amyloid fibrils. We propose that repositioning pyrvinium pamoate and apomorphine hydrochloride as TTR amyloid-disrupting agents may enable evaluation of their clinical utility for managing ATTR amyloidosis.

Academic Significance and Societal Importance of the Research Achievements

アミロイドーシスの根治には、アミロイドブレイカーの開発が極めて重要であるが、これまでATTRアミロイドーシスを培養細胞で再現することが出来なかった。本研究で確立したcell-based assay法は、アミロイドブレイカーのスクリーニング法として有用である。今後は、様々なライブラリーを用いてアミロイドブレイカーを探索する。アミロイド・ブレイカーの開発により、病態が進行した患者にも有効な治療法が提供できると考える。

Research Products

• [Journal Article] The prostate as a good candidate organ for proving amyloid deposition in non-endemic late onset hereditary transthyretin amyloidosis: Report of two cases (2021)
  • Author(s): Maeda Kentaro、Kubota Yasuaki、Kitagawa Satoshi、Ueda Mitsuharu、Ando Yukio、Ito Yasuhiro
  • Journal Title: Journal of the Neurological Sciences Volume: 424 Pages: 117418-117418
  • DOI: 10.1016/j.jns.2021.117418
  • Peer Reviewed / Int'l Joint Research
• [Journal Article] Current Management and Therapeutic Strategies for Cerebral Amyloid Angiopathy (2021)
  • Author(s): Yasuteru Inoue, Yukio Ando, Yohei Misumi, Mitsuharu Ueda
  • Journal Title: Int J Mol Sci Volume: 22 Issue: 8 Pages: 3869-3869
  • DOI: 10.3390/ijms22083869
  • Peer Reviewed / Open Access / Int'l Joint Research
• [Journal Article] Amyloid fibril formation is suppressed in microgravity (2021)
  • Author(s): Matsushita Hiroaki、Isoguchi Aito、Okada Masamitsu、Masuda Teruaki、Misumi Yohei、Ichiki Yuko、Ueda Mitsuharu、Ando Yukio
  • Journal Title: Biochemistry and Biophysics Reports Volume: 25 Pages: 100875-100875
  • DOI: 10.1016/j.bbrep.2020.100875
  • Peer Reviewed / Open Access
• [Journal Article] Reliability of the Modified Rankin Scale Assessment Using a Simplified Questionnaire in Japanese (2021)
  • Author(s): Yi Kenichiro、Inatomi Yuichiro、Nakajima Makoto、Yonehara Toshiro、Ueda Mitsuharu
  • Journal Title: Journal of Stroke and Cerebrovascular Diseases Volume: 30 Issue: 2 Pages: 105517-105517
  • DOI: 10.1016/j.jstrokecerebrovasdis.2020.105517
  • Peer Reviewed
• [Journal Article] Carpal Tunnel Syndrome Due to Iatrogenic Amyloidosis After Domino Liver Transplantation From Hereditary Transthyretin Amyloidosis: A Case Report (2021)
  • Author(s): Ohya Yuki、Tasaki Masayoshi、Hayashida Shintaro、Katayama Nobuhiro、Tsuchida Toru、Kuriwaki Kazumi、Ueda Mitsuharu、Inomata Yukihiro
  • Journal Title: Transplantation Proceedings Volume: － Issue: 4 Pages: 1313-1316
  • DOI: 10.1016/j.transproceed.2021.03.001
  • Peer Reviewed
• [Journal Article] Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study (2021)
  • Author(s): Adams David, et al.
  • Journal Title: The Lancet Neurology Volume: 20 Issue: 1 Pages: 49-59
  • DOI: 10.1016/s1474-4422(20)30368-9
  • Peer Reviewed / Int'l Joint Research
• [Journal Article] EGF-containing fibulin-like extracellular matrix protein 1 amyloid incidentally found in surgically resected specimens of Colon cancer: a case report emphasising on an unrecognised type (2021)
  • Author(s): Tsukamoto Yoshitane、Tasaki Masayoshi、Fujii Hitoshi、Tsujie Masaki、Ueda Mitsuharu
  • Journal Title: Amyloid Volume: 28 Issue: 2 Pages: 136-137
  • DOI: 10.1080/13506129.2020.1841624
  • Peer Reviewed
• [Journal Article] Plasma growth differentiation factor 15: a novel tool to detect early changes of hereditary transthyretin amyloidosis (2020)
  • Author(s): Okada Masamitsu、Misumi Yohei、Masuda Teruaki、Takashio Seiji、Tasaki Masayoshi、Matsushita Hiroaki、Ueda Akihiko、Inoue Yasuteru、Nomura Toshiya、Nakajima Makoto、Yamashita Taro、Shinriki Satoru、Matsui Hirotaka、Tsujita Kenichi、Ando Yukio、Ueda Mitsuharu
  • Journal Title: ESC Heart Failure Volume: 8 Issue: 2 Pages: 1178-1185
  • DOI: 10.1002/ehf2.13176
  • Peer Reviewed / Open Access
• [Journal Article] Anti-Kir4.1 Antibodies in Multiple Sclerosis: Specificity and Pathogenicity (2020)
  • Author(s): Imamura Michie、Higuchi Osamu、Maeda Yasuhiro、Mukaino Akihiro、Ueda Mitsuharu、Matsuo Hidenori、Nakane Shunya
  • Journal Title: International Journal of Molecular Sciences Volume: 21 Issue: 24 Pages: 9632-9632
  • DOI: 10.3390/ijms21249632
  • Peer Reviewed / Open Access
• [Journal Article] Usefulness of relative apical longitudinal strain index to predict positive 99m Tc‐labeled pyrophosphate scintigraphy findings in advanced‐age patients with suspected transthyretin amyloid cardiomyopathy (2020)
  • Author(s): Usuku Hiroki、Takashio Seiji、Yamamoto Eiichiro、Kinoshita Yui、Nishi Masato、Oike Fumi、Marume Kyohei、Hirakawa Kyoko、Tabata Noriaki、Oda Seitaro、Misumi Yohei、Ueda Mitsuharu、Kawano Hiroaki、Kaikita Koichi、Matsushita Kenichi、Ando Yukio、Matsui Hirotaka、Tsujita Kenichi
  • Journal Title: Echocardiography Volume: 37 Issue: 11 Pages: 1774-1783
  • DOI: 10.1111/echo.14892
  • Peer Reviewed
• [Journal Article] Clinical characteristics and natural history of wild‐type transthyretin amyloid cardiomyopathy in Japan (2020)
  • Author(s): Yamada Toshihiro、Takashio Seiji、Arima Yuichiro、Nishi Masato、Morioka Mami、Hirakawa Kyoko、Hanatani Shinsuke、Fujisue Koichiro、Yamanaga Kenshi、Kanazawa Hisanori、Sueta Daisuke、Araki Satoshi、Usuku Hiroki、Nakamura Taishi、Suzuki Satoru、Yamamoto Eiichiro、Ueda Mitsuharu、Kaikita Koichi、Tsujita Kenichi
  • Journal Title: ESC Heart Failure Volume: 7 Issue: 5 Pages: 2829-2837
  • DOI: 10.1002/ehf2.12884
  • Peer Reviewed / Open Access
• [Journal Article] Characteristics of Patients with Hereditary Transthyretin Amyloidosis and an Evaluation of the Safety of Tafamidis Meglumine in Japan: An Interim Analysis of an All-case Postmarketing Surveillance (2020)
  • Author(s): Ishii Tomonori、Hirano Yoko、Matsumoto Noriko、Takata Ami、Sekijima Yoshiki、Ueda Mitsuharu、Ando Yukio
  • Journal Title: Clinical Therapeutics Volume: 42 Issue: 9 Pages: 1728-1737.e6
  • DOI: 10.1016/j.clinthera.2020.07.001
  • Peer Reviewed / Open Access
• [Journal Article] Novel dot-blot assay for detection of vascular Notch3 aggregates in patients with CADASIL (2020)
  • Author(s): Ma Y, Ueda M, Ueda A, Shinriki S, Nagatoshi A, Isoguchi A, Okada M, Tasaki M, Nomura T, Inoue Y, Masuda T, Misumi Y, Yamashita T, Matsui H, Ando Y.
  • Journal Title: J Neurol Sci Volume: 415 Pages: 116931-116931
  • DOI: 10.1016/j.jns.2020.116931
  • Peer Reviewed
• [Journal Article] Monitoring of asymptomatic family members at risk of hereditary transthyretin amyloidosis for early intervention with disease-modifying therapies (2020)
  • Author(s): Ueda Mitsuharu、Sekijima Yoshiki、Koike Haruki、Yamashita Taro、Yoshinaga Tsuneaki、Ishii Tomonori、Ando Yukio
  • Journal Title: Journal of the Neurological Sciences Volume: 414 Pages: 116813-116813
  • DOI: 10.1016/j.jns.2020.116813
  • Peer Reviewed / Open Access
• [Journal Article] Trends in Diagnostic Imaging of Cardiac Amyloidosis: Emerging Knowledge and Concepts (2020)
  • Author(s): Oda Seitaro、Kidoh Masafumi、Nagayama Yasunori、Takashio Seiji、Usuku Hiroki、Ueda Mitsuharu、Yamashita Taro、Ando Yukio、Tsujita Kenichi、Yamashita Yasuyuki
  • Journal Title: RadioGraphics Volume: 40 Issue: 4 Pages: 961-981
  • DOI: 10.1148/rg.2020190069
  • Peer Reviewed
• [Journal Article] Human amyloidosis, still intractable but becoming curable: the essential role of pathological diagnosis in the selection of type-specific therapeutics. (2020)
  • Author(s): Naiki H, Sekijima Y, Ueda M, Ohashi K, Hoshii Y, Shimoda M, Ando Y.
  • Journal Title: Pathol Int Volume: 70 Issue: 4 Pages: 191-198
  • DOI: 10.1111/pin.12902
  • Peer Reviewed / Open Access
• [Journal Article] Integrated diagnostic approach to wild-type transthyretin cardiac amyloidosis with the use of high-sensitivity cardiac troponin T measurement and 99mTc-pyrophosphate scintigraphy. (2020)
  • Author(s): Ochi Y, Kubo T, Nakashima Y, Baba Y, Hirota T, Yamasaki N, Yamashita T, Ueda M, Ando Y, Kitaoka H.
  • Journal Title: J Cardiol Volume: 75 Issue: 1 Pages: 12-19
  • DOI: 10.1016/j.jjcc.2019.05.011
  • Peer Reviewed / Open Access
• [Journal Article] JCS 2020 Guideline on Diagnosis and Treatment of Cardiac Amyloidosis (2020)
  • Author(s): Kitaoka H, Izumi C, Izumiya Y, Inomata T, Ueda M, Kubo T, Koyama J, Sano M, Sekijima Y, Tahara N, Tsukada N, Tsujita K, Tsutsui H, Tomita T, Amano M, Endo J, Okada A, Oda S, Takashio S, Baba Y, Misumi Y, Yazaki M, Anzai T, Ando Y, Isobe M, Kimura T, Fukuda K; Japanese Circulation Society Joint Working Group.
  • Journal Title: Cir J Volume: 84 Issue: 9 Pages: 1610-1671
  • DOI: 10.1253/circj.cj-20-0110
  • NAID: 130007890677
  • Peer Reviewed / Open Access
• [Journal Article] Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis. (2020)
  • Author(s): Gonzalez-Duarte A, Berk J, Quan D, Mauermann M, Schmidt H, Polydefkis M, Waddington-Cruz M, Ueda M, Conceicao I, Kristen A, Coelho T, Cauquil C, Tard C, Merkel M, Aldinc E, Chen J, Sweetser M, Wang J, Adams D.
  • Journal Title: J Neurol Volume: 267 Issue: 3 Pages: 703-712
  • DOI: 10.1007/s00415-020-09715-5
  • Peer Reviewed / Int'l Joint Research
• [Journal Article] A Statement on the Appropriate Administration of Tafamidis in Patients With Transthyretin Cardiac Amyloidosis (2019)
  • Author(s): Endo Jin、Sano Motoaki、Izumiya Yasuhiro、Tsujita Kenichi、Nakamura Kazufumi、Tahara Nobuhiro、Kuwahara Koichiro、Inomata Takayuki、Ueda Mitsuharu、Sekijima Yoshiki、Ando Yukio、Tsutsui Hiroyuki、Isobe Mitsuaki、Fukuda Keiichi
  • Journal Title: Circulation Journal Volume: 84 Issue: 1 Pages: 15-17
  • DOI: 10.1253/circj.CJ-19-0811
  • NAID: 130007772713
  • ISSN: 1346-9843, 1347-4820
  • Year and Date: 2019-12-25
  • Peer Reviewed / Open Access / Int'l Joint Research
• [Journal Article] Hereditary ATTR Amyloidosis with Cardiomyopathy Caused by the Novel Variant Transthyretin Y114S (p.Y134S) (2019)
  • Author(s): Nakase Taku、Yamashita Taro、Matsuo Yoshimasa、Nomura Toshiya、Sasada Keiko、Masuda Teruaki、Misumi Yohei、Takamatsu Kotaro、Oda Seitaro、Furukawa Yutaro、Obayashi Konen、Matsui Hirotaka、Ando Yukio、Ueda Mitsuharu
  • Journal Title: Internal Medicine Volume: 58 Issue: 18 Pages: 2695-2698
  • DOI: 10.2169/internalmedicine.2456-18
  • NAID: 130007706923
  • ISSN: 0918-2918, 1349-7235
  • Year and Date: 2019-09-15
  • Peer Reviewed / Open Access
• [Journal Article] Reader response: Natural history and survival in stage 1 Val30Met transthyretin familial amyloid polyneuropathy (2019)
  • Author(s): Ueda Mitsuharu、Yamashita Taro、Misumi Yohei、Masuda Teruaki、Ando Yukio
  • Journal Title: Neurology Volume: 93 Issue: 5 Pages: 228-229
  • DOI: 10.1212/wnl.0000000000007870
  • Peer Reviewed
• [Journal Article] A cell-based high-throughput screening method to directly examine transthyretin amyloid fibril formation at neutral pH (2019)
  • Author(s): Ueda Mitsuharu、Okada Masamitsu、Mizuguchi Mineyuki、et al.
  • Journal Title: Journal of Biological Chemistry Volume: 294 Issue: 29 Pages: 11259-11275
  • DOI: 10.1074/jbc.ra119.007851
  • Peer Reviewed / Open Access / Int'l Joint Research
• [Journal Article] Natural history and long-term effects of variant protein reduction in non-V30M ATTR amyloidosis. (2019)
  • Author(s): Yamashita T, Ueda M, Nomura T, Okazaki T, Okada M, Tsuda Y, Inoue Y, Masuda T, Misumi Y, Takamatsu K, Obayashi K, Inomata Y, Hibi T, Ando Y.
  • Journal Title: Neurology Volume: 93 Issue: 16 Pages: 714-716
  • DOI: 10.1212/wnl.0000000000008320
  • Peer Reviewed
• [Journal Article] Nationwide survey on cerebral amyloid angiopathy in Japan. (2019)
  • Author(s): Sakai K, Ueda M, Fukushima W, Tamaoka A, Shoji M, Ando Y, Yamada M.
  • Journal Title: Eur J Neurol. Volume: 26(12) Issue: 12 Pages: 1487-1493
  • DOI: 10.1111/ene.14031
  • Peer Reviewed
• [Journal Article] Memantine, a noncompetitive N-methyl-D-aspartate receptor antagonist, attenuates cerebral amyloid angiopathy by increasing insulin-degrading enzyme expression. (2019)
  • Author(s): Inoue Y, Ueda M, Masuda M, Misumi Y, Yamashita T, Ando Y.
  • Journal Title: Mol Neurobiol Volume: 56 Issue: 12 Pages: 8573-8573
  • DOI: 10.1007/s12035-019-01678-7
  • Peer Reviewed
• [Journal Article] New simple and quick method to analyze serum variant transthyretins: direct MALDI method for the screening of hereditary transthyretin amyloidosis (2019)
  • Author(s): Nomura Toshiya、Ueda Mitsuharu、Tasaki Masayoshi、Misumi Yohei、Masuda Teruaki、Inoue Yasuteru、Tsuda Yukimoto、Okada Masamitsu、Okazaki Takahiro、Kanenawa Kyosuke、Isoguchi Aito、Nakamura Makoto、Obayashi Konen、Shinriki Satoru、Matsui Hirotaka、Yamashita Taro、Ando Yukio
  • Journal Title: Orphanet Journal of Rare Diseases Volume: 14 Issue: 1 Pages: 116-116
  • DOI: 10.1186/s13023-019-1100-y
  • Peer Reviewed / Open Access
• [Journal Article] Characteristics of acquired transthyretin amyloidosis (2019)
  • Author(s): Misumi Yohei、Ueda Mitsuharu、Masuda Teruaki、Tsuda Yukimoto、Nomura Toshiya、Okada Masamitsu、Inoue Yasuteru、Tasaki Masayoshi、Obayashi Konen、Yamashita Taro、Ando Yukio
  • Journal Title: Neurology Volume: － Issue: 17 Pages: 1587-1596
  • DOI: 10.1212/wnl.0000000000008360
  • Peer Reviewed
• [Journal Article] Early diagnosis of ATTR amyloidosis through targeted follow-up of identified carriers of TTR gene mutations* (2019)
  • Author(s): Conceicao Isabel、Damy Thibaud、Romero Manuel、Galan Lucia、Attarian Shahram、Luigetti Marco、Sadeh Menachem、Sarafov Stayko、Tournev Ivailo、Ueda Mitsuharu
  • Journal Title: Amyloid Volume: 26 Issue: 1 Pages: 3-9
  • DOI: 10.1080/13506129.2018.1556156
  • Peer Reviewed / Open Access / Int'l Joint Research
• [Journal Article] Apolipoprotein E and clusterin inhibit the early phase of amyloid-β aggregation in an in vitro model of cerebral amyloid angiopathy (2019)
  • Author(s): Endo Y, Hasegawa K, Nomura R, Arishima H, Kikuta K, Yamashita T, Inoue Y, Ueda M, Ando Y, Wilson MR, Hamano T, Nakamoto Y, Naiki H.
  • Journal Title: Acta Neuropathologica Communications Volume: 7 Issue: 1 Pages: 12-12
  • DOI: 10.1186/s40478-019-0662-1
  • Peer Reviewed / Open Access / Int'l Joint Research
• [Journal Article] A novel age-related venous amyloidosis derived from EGF-containing fibulin-like extracellular matrix protein 1 (2019)
  • Author(s): Tasaki M, Ueda M, Hoshii Y, Mizukami M, Matsumoto S, Nakamura M, Yamashita T, Ueda A, Misumi Y, Masuda T, Inoue Y, Torikai T, Nomura T, Tsuda Y, Kanenawa K, Isoguchi A, Okada M, Matsui H, Obayashi K, Ando Y.
  • Journal Title: The Journal of Pathology Volume: 247 Issue: 4 Pages: 444-455
  • DOI: 10.1002/path.5203
  • Peer Reviewed
• [Journal Article] First Nationwide Survey of 199 Patients with Amyloid A Amyloidosis in Japan (2018)
  • Author(s): Okuda Yasuaki、Yamada Toshiyuki、Ueda Mitsuharu、Ando Yukio
  • Journal Title: Internal Medicine Volume: 57 Issue: 23 Pages: 3351-3355
  • DOI: 10.2169/internalmedicine.1099-18
  • NAID: 130007539655
  • ISSN: 0918-2918, 1349-7235
  • Year and Date: 2018-12-01
  • Peer Reviewed / Open Access
• [Journal Article] Extreme Adhesion Activity of Amyloid Fibrils Induces Subcutaneous Insulin Resistance (2018)
  • Author(s): Nakamura M, Misumi Y, Nomura T, Oka W, Isoguchi A, Kanenawa K, Masuda T, Yamashita T, Inoue Y, Ando Y, Ueda M.
  • Journal Title: Diabetes Volume: 68 Issue: 3 Pages: 609-616
  • DOI: 10.2337/db18-0846
  • Peer Reviewed
• [Journal Article] Non-Val30Met mutation, septal hypertrophy, and cardiac denervation in patients with mutant transthyretin amyloidosis (2018)
  • Author(s): Hirakawa Kyoko、Takashio Seiji、Marume Kyohei、Yamamoto Masahiro、Hanatani Shinsuke、Yamamoto Eiichiro、Sakamoto Kenji、Izumiya Yasuhiro、Kaikita Koichi、Oda Seitaro、Utsunomiya Daisuke、Shiraishi Shinya、Ueda Mitsuharu、Yamashita Taro、Yamashita Yasuyuki、Ando Yukio、Tsujita Kenichi
  • Journal Title: ESC Heart Failure Volume: 6 Issue: 1 Pages: 122-130
  • DOI: 10.1002/ehf2.12361
  • Peer Reviewed / Open Access
• [Journal Article] Tongue strength in patients with subacute myelo-optico-neuropathy (2018)
  • Author(s): Yamashita Satoshi、Nakama Tatsuya、Ueda Mitsuharu、Honda Shoji、Kimura En、Konagaya Masaaki、Ando Yukio
  • Journal Title: Journal of Clinical Neuroscience Volume: 47 Pages: 84-88
  • DOI: 10.1016/j.jocn.2017.10.007
  • Peer Reviewed
• [Journal Article] Nationwide Survey of 741 Patients with Systemic Amyloid Light-chain Amyloidosis in Japan (2018)
  • Author(s): Shimazaki C, Hata H, Iida S, Ueda M, Katoh N, Sekijima Y, Ikeda S, Yazaki M, Fukushima W, Ando Y
  • Journal Title: Internal Medicine Volume: 57 Issue: 2 Pages: 181-187
  • DOI: 10.2169/internalmedicine.9206-17
  • NAID: 130006309174
  • ISSN: 0918-2918, 1349-7235
  • Peer Reviewed / Open Access
• [Journal Article] Loss of CYLD promotes cell invasion via ALK5 stabilization in oral squamous cell carcinoma (2018)
  • Author(s): Shinriki S, Jono H, Maeshiro M, Nakamura T, Guo J, Li JD, Ueda M, Yoshida R, Shinohara M, Nakayama H, Matsui H, Ando Y.
  • Journal Title: Journal of Pathology Volume: 244 Issue: 3 Pages: 367-379
  • DOI: 10.1002/path.5019
  • Peer Reviewed / Int'l Joint Research
• [Journal Article] Unwanted road to anemia in transthyretin familial amyloid polyneuropathy may continue irrespective of tafamidis treatment (2018)
  • Author(s): Ikeda T, Masuda T, Ueda M, Yamashita T, Misumi Y, Shinriki S, Ando Y.
  • Journal Title: Annals of Clinical Biochemistry Volume: 印刷中 Issue: 5 Pages: 571-575
  • DOI: 10.1177/0004563218754587
  • Peer Reviewed / Open Access
• [Journal Article] Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis (2018)
  • Author(s): Adams David、Gonzalez-Duarte Alejandra、O’Riordan William D.、Yang Chih-Chao、Ueda Mitsuharu, et al.
  • Journal Title: New England Journal of Medicine Volume: 379 Issue: 1 Pages: 11-21
  • DOI: 10.1056/nejmoa1716153
  • Peer Reviewed / Open Access / Int'l Joint Research
• [Journal Article] Teaching NeuroImages: Morphology of lumbosacral dorsal root ganglia and plexus in hereditary transthyretin amyloidosis (2018)
  • Author(s): Masuda T, Ueda M, Kitajima M, Morita K, Misumi Y, Yamashita T, Obayashi K, Yamashita Y, Ando Y
  • Journal Title: Neurology Volume: 91 Issue: 19
  • DOI: 10.1212/wnl.0000000000006474
  • Peer Reviewed / Open Access
• [Journal Article] Development of transgenic Caenorhabditis elegans expressing human transthyretin as a model for drug screening (2018)
  • Author(s): Tsuda Yukimoto、Yamanaka Kunitoshi、Toyoshima Risa、Ueda Mitsuharu、Masuda Teruaki、Misumi Yohei、Ogura Teru、Ando Yukio
  • Journal Title: Scientific Reports Volume: 8 Issue: 1 Pages: 17884-17884
  • DOI: 10.1038/s41598-018-36357-5
  • Peer Reviewed / Open Access
• [Journal Article] Diagnosis and management of transthyretin familial amyloid polyneuropathy in Japan: red-flag symptom clusters and treatment algorithm. (2018)
  • Author(s): Sekijima Y, Ueda M, Koike H, Misawa S, Ishii T, Ando Y
  • Journal Title: Orphanet J Rare Dis Volume: 13 Issue: 1 Pages: 6-6
  • DOI: 10.1186/s13023-017-0726-x
  • Peer Reviewed / Open Access
• [Journal Article] Origin of sporadic late-onset hereditary ATTR Val30Met amyloidosis in Japan (2018)
  • Author(s): Ueda Mitsuharu、Yamashita Taro、Misumi Yohei、Masuda Teruaki、Ando Yukio
  • Journal Title: Amyloid Volume: 25 Issue: 3 Pages: 143-147
  • DOI: 10.1080/13506129.2018.1531842
  • Peer Reviewed
• [Journal Article] A multicenter cross sectional study for bone-articular lesions associated with dialysis related amyloidosis in Japan. (2017)
  • Author(s): Nishi S, Hoshino J, Yamamoto S, Goto S, Fuji H, Ubara Y, Motomiya Y, Morita H, Takaichi K, Yamagata K, Shigematsu T, Ueda M, Ando Y
  • Journal Title: Nephrology Volume: 印刷中 Issue: 7 Pages: 640-645
  • DOI: 10.1111/nep.13077
  • Peer Reviewed / Open Access
• [Presentation] Development of amyloid disruptors for ATTR amyloidosis. (2020)
  • Author(s): Ueda M, Okada M, Mizuguchi M, Kluve-Beckerman B, Isoguchi A, Misumi Y, Tasaki M, Masuda T, Inoue Y, Nomura T, Obayashi K, Yamashita T, Benson M, Ando Y.
  • Organizer: XVII International Symposium on Amyloidosis.
  • Int'l Joint Research / Invited
• [Presentation] 遺伝性ATTRアミロイドーシス治療の現状と展望． (2020)
  • Author(s): 植田光晴
  • Organizer: 第38回日本神経治療学会学術集会
  • Invited
• [Presentation] Global Open-label Extension: 24-month Data of Patisiran in Patients with hATTR Amyloidosis. (2020)
  • Author(s): Ueda M, Adams D, Gonzalez-Duarte A, Mauricio E, Brannagan T, Coelho T, Wixner J, Schmidt H, Berber M, Sweetser M, White M, Wang J.J, Polydefkis M.
  • Organizer: 第38回日本神経治療学会学術集会
• [Presentation] 心不全パンデミックが迫っている中、今一度、hATTRアミロイドーシスについて考える！ (2019)
  • Author(s): 植田光晴
  • Organizer: 第83回日本循環器学会学術集会
  • Invited
• [Presentation] 遺伝性トランスサイレチン型アミロイドーシスの病態と治療． (2019)
  • Author(s): 植田光晴
  • Organizer: 第60回 日本神経学会学術大会
  • Invited
• [Presentation] 遺伝性ATTRアミロイドーシスの病態解析とsiRNA治療の可能性． (2019)
  • Author(s): 植田光晴
  • Organizer: 第7回 日本アミロイドーシス研究会
  • Invited
• [Presentation] Monitoring carriers of a TTR mutation. (2019)
  • Author(s): Ueda M.
  • Organizer: Advances and Research in TTR Amyloidosis
  • Int'l Joint Research / Invited
• [Presentation] Early skin denervation in transthyretin amyloid neuropathy. (2019)
  • Author(s): Ueda M.
  • Organizer: Advances and Research in TTR Amyloidosis
  • Int'l Joint Research / Invited
• [Presentation] Clinicopathological and biochemical studies on amyloidosis in the Kumamoto University Amyloidosis Center. (2019)
  • Author(s): Ueda M.
  • Organizer: Joint Symposium with Molecular Neurodegeneration
  • Int'l Joint Research / Invited
• [Presentation] Early diagnosis and therapies for hereditary transthyretin amyloidosis. (2018)
  • Author(s): Ueda M.
  • Organizer: XVI International Symposium on Amyloidosis
  • Int'l Joint Research / Invited
• [Presentation] Redefining the patient and treatment paradigm. (2018)
  • Author(s): Ueda M, Masuda T, Misumi Y, Yamashita T, Ando Y.
  • Organizer: XVI International Symposium on Amyloidosis
  • Int'l Joint Research / Invited
• [Presentation] Red-Flag symptoms for diagnosis of aTTR-PN patients (with focus on non-V30M). (2018)
  • Author(s): Ueda M.
  • Organizer: aTTR-PN/CM Advisory Board Meeting.
  • Int'l Joint Research / Invited
• [Book] 21世紀の疾患：神経関連アミロイドーシス (2020)
  • Author(s): 安東 由喜雄 (監修), 植田 光晴 (編集), 山下 太郎 (編集)
  • Total Pages: 135
  • Publisher: 医学と看護社
  • ISBN: 4909888071
• [Patent(Industrial Property Rights)] アミロイドーシス治療薬の新規スクリーニング方法及びアミロイドーシスのための医薬組成物 (2019)
  • Inventor(s): 植田光晴; 安東由喜雄; 水口峰之
  • Industrial Property Rights Holder: 植田光晴; 安東由喜雄; 水口峰之
  • Industrial Property Rights Type: 特許
  • Filing Date: 2019