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Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome

http://hdl.handle.net/20.500.12000/45879
http://hdl.handle.net/20.500.12000/45879
c4fbdbcf-48c0-47e0-a73c-d74861ed3240
名前 / ファイル ライセンス アクション
DDDT-68562-impact-of-enzyme-replacement-therapy-and-hematopoietic-stem-_040115.pdf DDDT-68562-impact-of-enzyme-replacement-therapy-and-hematopoietic-stem-_040115.pdf
Item type デフォルトアイテムタイプ(フル)(1)
公開日 2020-05-22
タイトル
タイトル Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome
言語 en
作成者 Tomatsu, Shunji

× Tomatsu, Shunji

en Tomatsu, Shunji

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Sawamoto, Kazuki

× Sawamoto, Kazuki

en Sawamoto, Kazuki

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Almeciga-Diaz, Carlos J.

× Almeciga-Diaz, Carlos J.

en Almeciga-Diaz, Carlos J.

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Shimada, Tsutomu

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en Shimada, Tsutomu

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Bober, Michael B.

× Bober, Michael B.

en Bober, Michael B.

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Chinen, Yasutsugu

× Chinen, Yasutsugu

en Chinen, Yasutsugu

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Yabe, Hiromasa

× Yabe, Hiromasa

en Yabe, Hiromasa

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Montano, Adriana M.

× Montano, Adriana M.

en Montano, Adriana M.

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Giugliani, Roberto

× Giugliani, Roberto

en Giugliani, Roberto

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Kubaski, Francyne

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en Kubaski, Francyne

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Yasuda, Eriko

× Yasuda, Eriko

en Yasuda, Eriko

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Rodriguez-Lopez, Alexander

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en Rodriguez-Lopez, Alexander

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Espejp-Mojica, Angela J.

× Espejp-Mojica, Angela J.

en Espejp-Mojica, Angela J.

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Sanchez, Oscar F.

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en Sanchez, Oscar F.

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Mason, Robert W.

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Barrera, Luis A.

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en Barrera, Luis A.

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Mackenize, William G.

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Orii, Tadao

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アクセス権
アクセス権 open access
アクセス権URI http://purl.org/coar/access_right/c_abf2
権利情報
言語 en
権利情報 Creative Commons Attribution-NonCommercial 4.0
権利情報
言語 en
権利情報Resource https://creativecommons.org/licenses/by-nc/4.0/
権利情報 https://creativecommons.org/licenses/by-nc/4.0/
主題
言語 en
主題Scheme Other
主題 mucopolysaccharidosis IVA
主題
言語 en
主題Scheme Other
主題 ERT
主題
言語 en
主題Scheme Other
主題 HSCT
主題
言語 en
主題Scheme Other
主題 skeletal dysplasia
主題
言語 en
主題Scheme Other
主題 keratan sulfate
内容記述
内容記述タイプ Other
内容記述 Patients with mucopolysaccharidosis IVA (MPS IVA) can present with systemic skeletal dysplasia, leading to a need for multiple orthopedic surgical procedures, and often become wheelchair bound in their teenage years. Studies on patients with MPS IVA treated by enzyme replacement therapy (ERT) showed a sharp reduction on urinary keratan sulfate, but only modest improvement based on a 6-minute walk test and no significant improvement on a 3-minute climb-up test and lung function test compared with the placebo group, at least in the short-term. Surgical remnants from ERT-treated patients did not show reduction of storage materials in chondrocytes. The impact of ERT on bone lesions in patients with MPS IVA remains limited. ERT seems to be enhanced in a mouse model of MPS IVA by a novel form of the enzyme tagged with a bone-targeting moiety. The tagged enzyme remained in the circulation much longer than untagged native enzyme and was delivered to and retained in bone. Three-month- old MPS IVA mice treated with 23 weekly infusions of tagged enzyme showed marked clearance of the storage materials in bone, bone marrow, and heart valves. When treatment was initiated at birth, reduction of storage materials in tissues was even greater. These findings indicate that specific targeting of the enzyme to bone at an early stage may improve efficacy of ERT for MPS IVA. Recombinant N-acetylgalactosamine-6-sulfate sulfatase (GALNS) in Escherichia coli BL21 (DE3) (erGALNS) and in the methylotrophic yeast Pichia pastoris (prGALNS) has been produced as an alternative to the conventional production in Chinese hamster ovary cells. Recombinant GALNS produced in microorganisms may help to reduce the high cost of ERT and the introduction of modifications to enhance targeting. Although only a limited number of patients with MPS IVA have been treated with hematopoietic stem cell transplantation (HSCT), beneficial effects have been reported. A wheelchair-bound patient with a severe form of MPS IVA was treated with HSCT at 15 years of age and followed up for 10 years. Radiographs showed that the figures of major and minor trochanter appeared. Loud snoring and apnea disappeared. In all, 1 year after bone marrow transplantation, bone mineral density at L2-L4 was increased from 0.372 g/cm(2) to 0.548 g/cm(2) and was maintained at a level of 0.48 +/- 0.054 for the following 9 years. Pulmonary vital capacity increased approximately 20% from a baseline of 1.08 L to around 1.31 L over the first 2 years and was maintained thereafter. Activity of daily living was improved similar to the normal control group. After bilateral osteotomies, a patient can walk over 400 m using hip-knee-ankle-foot orthoses. This long-term observation of a patient shows that this treatment can produce clinical improvements although bone deformity remained unchanged. In conclusion, ERT is a therapeutic option for MPS IVA patients, and there are some indications that HSCT may be an alternative to treat this disease. However, as neither seems to be a curative therapy, at least for the skeletal dysplasia in MPS IVA patients, new approaches are investigated to enhance efficacy and reduce costs to benefit MPS IVA patients.
内容記述
内容記述タイプ Other
内容記述 論文
出版者
出版者 Dove Medical Press
言語 en
言語
言語 eng
資源タイプ
資源タイプ識別子 http://purl.org/coar/resource_type/c_6501
資源タイプ journal article
出版タイプ
出版タイプ VoR
出版タイプResource http://purl.org/coar/version/c_970fb48d4fbd8a85
識別子
識別子 http://hdl.handle.net/20.500.12000/45879
識別子タイプ HDL
関連情報
識別子タイプ DOI
関連識別子 https://doi.org/10.2147/DDDT.S68562
関連情報
識別子タイプ DOI
関連識別子 https://doi.org/10.2147/DDDT.S68562
収録物識別子
収録物識別子タイプ ISSN
収録物識別子 1177-8881
収録物名
収録物名 Drug Design, Development and Therapy
言語 en
書誌情報
巻 9, p. 1937-1953, 発行日 2015-04-01
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