top of page
Library Shelves_edited_edited.jpg

研　究　業　績

  • 松本 多絵：低ホスファターゼ症モデルマウスにおける遺伝子治療 ―臨床応用にむけて―。日本医科大学医学会雑誌 19: 229-234, 2023.

 

 

 

​

  • 三宅 弘一：低ホスファターゼ症に対する遺伝子治療。 Precision Medicine 6: 226-230, 2023.

 

  • 三宅 弘一：低ホスファターゼ症に対する新規治療法（遺伝子治療薬）の開発。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)

​

  • 三宅 弘一：低ホスファターゼ症に対する新規治療法(遺伝子治療薬）の開発。 別冊 BIO Clinica 11: 92-96, 2022.

​​

  • 三宅 弘一：低ホスファターゼ症の新規治療法(遺伝子治療薬：ARU-2801）の開発。 Precision medicine 5: 944-949, 2022.

​​

  • 三宅 弘一：低ホスファターゼ症に対する遺伝子治療薬（ARU-2801）の開発。 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)

  • 三宅弘一, 三宅紀子: Development of adeno-associated virus vector-mediated gene therapy for inherited diseases. SNBL Science Seminar, 2023年12月5日, Gyeonggi-do, 韓国

 

  • 松本 多絵, 趙 東威, 三宅 紀子, 成澤 園子, Millán Luis José, 三宅 弘一: 低ホスファターゼ症に対する新規治療薬（遺伝子治療薬：ARU-2801）の有効性と安全性の検討（シンポジウム：Genetic Diseases）. The 29th Annual Meeting of Japan Society of Gene Therapy, September 11-13, 2023, 大阪.

​

  • 松本多絵, 三宅紀子, 趙東威, 田中悠介, Mohammad Shadid, 成澤園子, 折茂英生, 島田隆, José Luis Millán, 三宅弘一: 低ホスファターゼ症に対する新規治療薬（遺伝子治療薬：ARU-2801）の有効性の検討. 第15回ALPS研究会, 2023年7月8日, 鶴見

 

  • 趙東威, 松本多絵, 三宅紀子, 田中悠介, Mohammad Shadid, 成澤園子, 折茂英生, 島田隆, José Luis Millán, 三宅弘一: 低ホスファターゼ症に対する新規治療薬（遺伝子治療薬：ARU-2801）の安全性の検討. 第15回ALPS研究会, 2023年7月8日, 鶴見

​

  • 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.

​

  • 松本 多絵, 三宅 紀子, 趙 東威, Mohammad Shadid, Eric Gaukel, 成澤 園子, Millán Luis José, 三宅 弘一：低ホスファターゼ症に対する新規治療法（遺伝子治療薬​：ARU-2801）の開発. 日本人類遺伝学会第67回大会, 2022年12月14‐17, 横浜

 

  • 松本 多絵, 三宅 紀子, 趙 東威, Mohammad Shadid, Eric Gaukel, 成澤 園子, Millán Luis José, 三宅 弘一：低ホスファターゼ症に対する新規治療法（遺伝子治療薬）の開発. 第55回日本小児内分泌学会学術集会, 2022年11月1-3日, 横浜

 

  • 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.

 

  • 三宅 弘一：遺伝子治療薬開発の現状と課題―毒性を中心にー. 第8回浜松毒性試験フォーラム, 2022年10月28日, 浜松

 

  • 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.

​

  • 趙 東威, 松本 多絵, 三宅 紀子, Shadid Mohammad, Gaukel Eric, 成澤 園子, Millán Luis José, 三宅 弘一：低ホスファターゼ症に対する遺伝子治療薬（ARU-2801）の有効性及び安全性の検討. The 28th Annual Meeting of Japan Society of Gene Therapy, 2022年7月14-16日, 福岡

 

  • 三宅 紀子, 三宅 弘一, 酒井 真志人, 島田 隆：異染性白質ジストロフィーの遺伝子治療（シンポジウム：神経・筋疾患）. The 28th Annual Meeting of Japan Society of Gene Therapy, 2022年7月14-16日, 福岡

 

  • 三宅 弘一：遺伝子治療薬における安全性評価の現状と課題（シンポジウム：遺伝子・細胞治療薬開発とその安全性評価）. 第49回日本毒性学会学術年会, 2022年6月30日-7月2日, 札幌

 

  • 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.

社会連携講座発足以前の低ホスファターゼ症に対する遺伝子治療関係の論文

  • 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)

bottom of page