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Publications

  • Matsumoto T:Gene Therapy for Hypophosphatasia in Alpl-/- mice (infantile HPP model) ―Toward Clinical Application―. The medical Association of Nippon Medical School. 19: 229-234, 2023.

 

 

 

  • Miyake K:Gene therapy for Hypophosphatasia. Precision medicine 6: 266-230, 2023.

 

  • Miyake K:Development of a novel gene therapy drug for Hypophosphatasia. BIO Clinica 38: 87-91, 2023.

 

  • Amadeu de Oliveira F, Mohamed FF, Kinoshita Y, Narisawa S, Farquharson C, Miyake K, Foster BL, Millán JL: Gene Therapy Using Recombinant AAV Type 8 Vector Encoding TNAP-D10 Improves the Skeletal Phenotypes in Murine Models of Osteomalaci. JBMR Plus. 7: e10709, 2022. (doi: 10.1002/jbm4.10709)

  • Miyake K:Development of a novel gene therapy drug for Hypophosphatasia. BIO Clinica (a separate volume) 11: 92-96, 2022.

 

  • Miyake K:Development of a novel gene therapy drug (ARU-2801) for Hypophosphatasia. Precision medicine 5: 944-949, 2022.

 

  • Miyake K:Development gene therapy drug (ARU-2801) for Hypofosfatasia. BIO Clinica 37: 590-594, 2022.

 

  • Miyake N, Miyake K, Sakai A, Yamamoto M, Suzuki H, Shimada T: Treatment of adult metachromatic leukodystrophy model mice using intrathecal administration of type 9 AAV vector encoding arylsulfatase A. Sci Rep.11: 20513, 2021. (doi:10.1038/s41598-021-99979-2)

 

  • Miyake K, Miyake N, Shimada T: A New Method for In Vivo Targeted Gene Transfer into Oligodendrocytes using Adenoviral and HIV Vectors. Biomed J of Sci & Tech Res. 39: 31379-31385, 2021. (doi:10.26717/BJSTR.2021. 39.006306)

  • Kinoshita Y, Mohamed FF, Amadeu de Oliveira F, Narisawa S, Miyake K, Foster BL, Millán JL: Gene Therapy Using Adeno-Associated Virus Serotype 8 Encoding TNAP-D10 Improves the Skeletal and Dentoalveolar Phenotypes in Alpl-/- Mice. J Bone Miner Res. 36: 1835-1849, 2021. (doi:10.1002/jbmr.4382)

 

  • Matsumoto T, Miyake K, Miyake N, Iijima O, Adachi K, Orimo H, Narisawa S, Millán JL, Fukunaga Y, Shimada T: Treatment with bone maturation and average lifespan of HPP model mice by AAV8-mediated neonatal gene therapy via single muscle injection. Mol Ther Methods Clin Dev. 22: 330-337, 2021. (doi:10.1016/j.omtm. 2021.06.006)

  • Miyake K, Miyake N: Development of adeno-associated virus vector-mediated gene therapy for inherited diseases. SNBL Science Seminar, December 5, 2023, Gyeonggi-do, South Korea.

 

  • Matsumoto T, Zhao D, Miyake N, Narisawa S, Millán JL, Miyake K: Efficacy and Safety of a Novel Therapeutic Agent (Gene Therapy drug: ARU-2801) for the Treatment of Hypophosphatasia. The 29th Annual Meeting of Japan Society of Gene Therapy, September 11-13, 2023, Osaka, JPN.

  • Matsumoto T, Miyake N, Zhao D, Tanaka S, Shadid M, Narisawa S, Orimo H, Shimada T, Millán JL, Miyake K: Efficacy of a Novel Therapeutic Agent (Gene Therapy drug: ARU-2801) for the Treatment of Hypophosphatasia. The 15th ALPS Research Conference, July 8, 2023, Tsurumi, JPN.

 

  • Zhao D, Matsumoto T, Miyake N, Tanaka S, Shadid M, Narisawa S, Orimo H, Shimada T, Millán JL, Miyake K: Safety of a Novel Therapeutic Agent (Gene Therapy drug: ARU-2801) for the Treatment of Hypophosphatasia. The 15th ALPS Research Conference, July 8, 2023, Tsurumi, JPN.

  • Matsumoto T, Zhao D, Miyake N, Narisawa S, Millán JL, Miyake K: Development of gene therapy drug (ARU-2801) for HPP. 2nd Soft Bones International Scientific Meeting 2023, June 2-4, 2023 Bethesda, MD, USA.

 

  • Sugano H, Matsumoto T, Narisawa S, Millán JL, Miyake K: Possibility of prenatal gene therapy for lethal HPP model mice. 2nd Soft Bones International Scientific Meeting 2023, June 2-4, 2023 Bethesda, MD, USA.

 

  • Matsumoto T, Zhao D, Miyake N, Shadid M, Gaukel E, Narisawa S, Millán JL, Miyake K: Efficacy and safety of preclinical gene therapy for hypophosphatasia using ARU-2801 (AAV8 vector expressing TNALP-D10). The American Society for Gene and Cell Therapy (ASGCT) 26th Annual Meeting, May 16-20, 2023 Los Angeles, CA, USA.

  • Matsumoto T, Miyake N, Zhao D, Shadid M, Gaukel E, Narisawa S, Millán JL, Miyake K: Development of effective and safe gene therapy drug (ARU-2801) for hypophosphatasia. The 67th Annual Meeting of the Japan Society of Human Genetics, December 14-17, 2022, Yokohama, JPN.

 

  • Shadid M, Gaukel E, Zhao D, Miyake N, Tanaka Y, Matsumoto T, Miyake K: Evaluation of efficacy and safety of a novel gene therapy drug (ARU-2801) for hypophosphatasia in non-human primates. Society for Endocrinology BES 2022, November 14-16, 2022. Harrogate, UK.

  • Matsumoto T, Miyake N, Zhao D, Shadid M, Gaukel E, Narisawa S, Millán JL, Miyake K: Development of an adeno-associated viral gene therapy drug (ARU-2801) intended for hypophospnatasia. The 55th Annual Scientific Meeting of the Japanese Society for Pediatric Endocrinology, November 1-3, 2022, Yokohama, JPN.

 

  • Miyake K: Current Status and Issues in Gene Therapy Drug Development - Focusing on Toxicity-. The 8th Annual Meeting of HAMAMATSU TOXICOLOGY STUDY FORUM, October 28, 2022, Hamamatsu, JPN.

 

  • Shadid M, Gaukel E, Zhao D, Tanaka Y, Miyake N, Matsumoto T, Miyake K: Validity and safety preclinical in vivo study of gene therapy for hypophosphatasia using ARU-2801 (AAV8 vector expressing TNALP-D10). The 29th Annual Meeting of the European Society of Gene & Cell Therapy (ESGCT 2022), October 11-14, 2022, Edinburgh, Scotland.

  • Shadid M, Gaukel E, Zhao D, Miyake N, Matsumoto T, Miyake K: Non-human primate in vivo study of ARU-2801, an investigational adeno-associated viral gene therapy intended for hypophosphatasia, shows durable transgenic plasma alkaline phosphatase levels are achievable without any toxicities. The American Society for Bone and Mineral Research (ASBMR) 2022 Annual Meeting, September 9-12, 2022, Austin, TX, USA.

 

  • Amadeu de Oliveira F, Mohamed FF, Kinoshita Y, Narisawa S, Miyake K, Foster BL, Millán JL: Gene therapy using recombinant AAV serotype 8 encoding TNAP-D10 improves the skeletal phenotype in a late-onset HPP mouse model. The American Society for Bone and Mineral Research (ASBMR) 2022 Annual Meeting, September 9-12, 2022, Austin, TX, USA.

 

  • Amadeu de Oliveira F, Mohamed FF, Narisawa S, Kinoshita Y, Farquharson C, Miyake K, Foster BL, Millán JL: Gene therapy using recombinant AAV serotype 8 encoding TNAP-D10 improves the skeletal phenotype in the PHOSPHO1 deficient mouse model. The American Society for Bone and Mineral Research (ASBMR) 2022 Annual Meeting, September 9-12, 2022, Austin, TX, USA.

  • Zhao D, Matsumoto T, Miyake N, Mohammad S, Eric G, Narisawa S, Millán JL, Miyake K:Validity and safety preclinical in vivo study of gene therapy for hypophosphatasia using ARU-2801 (AAV8 vector expressing TNALP-D10). The 28th Annual Meeting of Japan Society of Gene Therapy, July 14-16, 2022Fukuoka, JPN.

 

  • Miyake N, Miyake K Sakai M, Shimada T: Gene therapy of metachromatic leukodystrophy(Symposium: Neuromuscular disease. The 28th Annual Meeting of Japan Society of Gene Therapy, July 14-16, 2022, Fukuoka, JPN.

 

  • Miyake K: Current Status and Issues of Safety Evaluation of Gene Therapy Drugs (Symposium: Development of Gene/Cell Therapy Drugs and Their Safety Evaluation). The 49th Annual Meeting of the Japanese Society of Toxicology, June 30-July 2, 2022, Sapporo, JPN.

 

  • Miyake N, Sakai A, Yamamoto M, Miyake K: Safety and efficacy of intrathecal administration of type 9 AAV vector encoding arylsulfatase A in adult metachromatic leukodystrophy model mice. 25th Annual Meeting of the American Society of Gene & Cell Therapy, May 16-19, 2022, Washington DC, USA.

 

  • Matsumoto T, Miyake N, Zhao D, Narisawa S, Millán JL, Miyake K: Successful adeno-associated virus mediated neonatal gene therapy treatment of hypophosphatasia murine model resulted in bone maturation and increased survival to at least 18 months. Society for Endocrinology BES 2021, December 8-10, 2021. Edinburgh, UK.

 

  • Kinoshita Y, Mohamed FF, Amadeu de Oliveira F, Narisawa S, Miyake K, Foster BL, Millán JL: Adeno-associated virus TNAP-D10-mediated gene therapy improves skeletal and dentoalveolar phenotypes in Alpl-/- mice. The American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting, October 1-4, 2021, San Diego, California, USA.

 

  • Matsumoto T, Miyake K, Miyake N, Narisawa S, Millán JL: Successful adeno-associated virus mediated neonatal gene therapy treatment of a murine model of infantile hypophosphatasia resulted in bone maturation and increased survival to at least 18 months. The American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting, October 1-4, 2021, San Diego, California, USA.

Papers related to gene therapy for hypophosphatasia before the establishment of the Department of Gene therapy

  • Yamamoto S, Orimo H, Matsumoto T, Iijima O, Narisawa S, Maeda T, Millán JL, Shimada T: Prolonged survival and phenotypic correction of Akp2(-/-) hypophosphatasia mice by lentiviral gene therapy. J Bone Miner Res, 2011; 26:135-42. (doi:10.1002/jbmr.201)

 

  • Matsumoto T, Miyake K, Yamamoto S, Orimo H, Miyake N, Odagaki Y, Adachi K, Iijima O, Narisawa S, Millán JL, Fukunaga Y, Shimada T: Rescue of severe infantile hypophosphatasia mice by AAV-mediated sustained expression of soluble alkaline phosphatase. Hum Gene Ther, 2011; 22: 1355-64. (doi:10.1089/hum.2010.210)

 

  • Sugano H, Matsumoto T, Miyake K, Watanabe A, Iijima O, Migita M, Narisawa S, Millán JL, Fukunaga Y, Shimada T: Successful gene therapy in utero for lethal murine hypophosphatasia. Hum Gene Ther, 2012; 23: 399-406. (doi:10.1089/hum.2011.148)

 

  • Iijima O, Miyake K, Watanabe A, Miyake N, Igarashi T, Kanokoda C, Nakamura-Takahashi A, Kinoshita H, Noguchi T, Abe S, Narisawa S, Millán JL, Okada T, Shimada T: Prevention of lethal murine hypophosphatasia by neonatal ex vivo gene therapy using lentivirally transduced bone marrow cells. Hum Gene Ther, 2015; 26: 801-12. (doi:10.1089/hum.2015.078)

 

  • Nakamura-Takahashi A, Miyake K, Watanabe A, Hirai Y, Iijima O, Miyake N, Adachi K, Nitahara-Kasahara Y, Kinoshita H, Noguchi T, Abe S, Narisawa S, Millán JL, Shimada T, Okada T: Treatment of hypophosphatasia by muscle-directed expression of bone-targeted alkaline phosphatase via self-complementary AAV8 vector. Mol Ther Methods Clin Dev, 2016; 3, 15059. (doi:10.1038/mtm.2015.59)

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