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研　究　成　果

  • 松本 多绘：《低磷酸酯酶症模型小鼠的基因治疗-- 面向临床应用》日本医科大学医学会杂志 19: 229-234, 2023.

 

 

 

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  • 三宅 弘一：《针对低磷酸酯酶症的基因治疗》 Precision Medicine 6: 226-230, 2023.

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  • 三宅 弘一：《低磷酸酯酶症新型治疗方法（基因治疗药物）的开发》 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).

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  • 三宅 弘一：《低磷酸酯酶症新型治疗方法（基因治疗药物）的开发》 别册 BIO Clinica 11: 92-96, 2022.

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  • 三宅 弘一：《低磷酸酯酶症新型治疗方法（基因治疗药物：ARU-2801）的开发》 Precision medicine 5: 944-949, 2022.

 

  • 三宅 弘一：《低磷酸酯酶症基因治疗药物（ARU-2801）的研发》 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)

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  • 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 纪子：《Development of adeno-associated virus vector-mediated gene therapy for inherited diseases. SNBL Science Seminar, 2023年12月5日, 韩国京畿道

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  • 松本 多绘, 赵 东威, 三宅 纪子, 成泽 园子, Millán Luis José, 三宅 弘一：《关于新型低磷酸酯酶症治疗药物（基因治疗药物：ARU-2801）的有效性和安全性（专题：Genetic Disease）》The 29th Annual Meeting of Japan Society of Gene Therapy, 2023年9月11-13日, 日本大阪

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  • 松本 多绘, 三宅 纪子, 赵 东威, 田中 悠介, Mohammad Shadid, 成泽 园子, 折茂 英生, 岛田 隆, Millán Luis José, 三宅 弘一：《关于新型低磷酸酯酶症治疗药物（基因治疗药物：ARU-2801）的有效性》第15回日本ALPS研究会, 2023年7月8日, 日本鹤见

 

  • 赵 东威, 松本 多绘, 三宅 纪子, 田中 悠介, Mohammad Shadid, 成泽 园子, 折茂 英生, 岛田 隆, Millán Luis José, 三宅 弘一：《关于新型低磷酸酯酶症治疗药物（基因治疗药物：ARU-2801）的安全性》第15回日本ALPS研究会, 2023年7月8日, 日本鹤见

 

  • 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.

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  • 松本 多绘, 赵 东威, 三宅 纪子, Shadid Mohammad, Gaukel Eric, Millán Luis José, 三宅 弘一：《低磷酸酯酶症新型治疗方法（基因治疗药物：ARU-2801）的开发》日本人类遗传学会第67回大会, 2022年12月14-17日, 日本横滨

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  • 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.

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  • 松本 多绘, 赵 东威, 三宅 纪子, Shadid Mohammad, Gaukel Eric, Millán Luis José, 三宅 弘一：《低磷酸酯酶症新型治疗方法（基因治疗药物：ARU-2801）的开发》第55回日本小儿内分泌学会学术年会, 2022年11月1-3日, 日本横滨

 

  • 三宅 弘一：《基因治疗药物开发的现状与课题 -聚焦于毒性-》参加日本第8回滨松毒性试验研讨会, 2022年10月28日, 日本滨松

 

  • 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.

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  • 赵 东威, 松本 多绘, 三宅 纪子, Shadid Mohammad, Gaukel Eric, 成泽 园子, Millán Luis José, 三宅 弘一：《针对低磷酸酯酶症基因治疗药物的有效性和安全性的相关实验》 The 28th Annual Meeting of Japan Society of Gene Therapy, 2022年7月14-16日, 日本福冈

 

  • 三宅 纪子, 三宅 弘一, 酒井 真志人, 岛田 隆：《异染性脑白质营养不良（MLD）的基因治疗（主题演讲：神经・肌肉疾病）》 The 28th Annual Meeting of Japan Society of Gene Therapy, 2022年7月14-16日, 日本福冈

 

  • 三宅 弘一：《关于基因治疗药物的现状和课题（主题演讲：关于基因・细胞治疗药物开发的安全性评价）》 第49回日本毒性学会学术年会, 2022年6月30日-7月2日. 日本札幌

 

  • 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.

联合研究室成立以前发表的有关低磷酸酯酶症基因治疗的论文

  • 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)

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