リケラボ論文検索は、全国の大学リポジトリにある学位論文・教授論文を一括検索できる論文検索サービスです。

リケラボ 全国の大学リポジトリにある学位論文・教授論文を一括検索するならリケラボ論文検索大学・研究所にある論文を検索できる

リケラボ 全国の大学リポジトリにある学位論文・教授論文を一括検索するならリケラボ論文検索大学・研究所にある論文を検索できる

大学・研究所にある論文を検索できる 「A new immunodeficient Duchenne muscular dystrophy rat model to evaluate engraftment after human cell transplantation」の論文概要。リケラボ論文検索は、全国の大学リポジトリにある学位論文・教授論文を一括検索できる論文検索サービスです。

コピーが完了しました

URLをコピーしました

論文の公開元へ論文の公開元へ
書き出し

A new immunodeficient Duchenne muscular dystrophy rat model to evaluate engraftment after human cell transplantation

Sato, Masae Goto, Megumi Yamanouchi, Keitaro Sakurai, Hidetoshi 京都大学 DOI:10.3389/fphys.2023.1094359

2023

概要

Duchenne muscular dystrophy (DMD) is an X-linked fatal muscular disease, affecting one in 3, 500 live male births worldwide. Currently, there is no cure for this disease, except for steroid-based treatment to attenuate disease progression. Cell transplantation therapy is a promising therapeutic approach, however, there is a lack of appropriate animal models to conduct large-scale preclinical studies using human cells, including biochemical and functional tests. Here, we established an immunodeficient DMD rat model and performed exhaustive pathological analysis and transplantation efficiency evaluation to assess its suitability to study DMD. Our DMD rat model exhibited histopathological characteristics similar to those observed in human patients with DMD. Human myoblasts demonstrated successful engraftment following transplantation into these rats. Therefore, this immunodeficient DMD rat model would be useful in preclinical studies to develop cellular transplantation therapies for DMD.

この論文で使われている画像

参考文献

Arpke, R. W., Darabi, R., Mader, T. L., Zhang, Y., Toyama, A., Lonetree, C. L.,

et al. (2013). A new immuno-dystrophin-deficient model, the NSG-mdx(4Cv)

mouse, provides evidence for functional improvement following allogeneic

satellite cell transplantation. Stem Cells. 31 (8), 1611–1620. doi:10.1002/stem.1402

Marg, A., Escobar, H., Gloy, S., Kufeld, M., Zacher, J., Spuler, A., et al. (2014). Human

satellite cells have regenerative capacity and are genetically manipulable. J. Clin.

Investigation 124 (10), 4257–4265. doi:10.1172/JCI63992

Maruyama, N., Asai, T., Abe, C., Inada, A., Kawauchi, T., Miyashita, K., et al. (2016).

Establishment of a highly sensitive sandwich ELISA for the N-terminal fragment of titin

in urine. Sci. Rep. 6, 39375. doi:10.1038/srep39375

Beytía, M. A., Vry, J., and Kirschner, J. (2012). Drug treatment of duchenne muscular

dystrophy: Available evidence and perspectives. Acta Myol. 31, 4–8.

Cao, X., Shores, E. W., Hu-Li, J., Anver, M. F., Kelsall, B. L., Rssell, S. M., et al. (1995).

Defective lymphoid development in mice lacking expression of the common cytokine

receptor γ chain. Immunity 2, 223–238.

Miura, Y., Sato, M., Kuwahara, T., Ebata, T., Tabata, Y., Sakurai, H., et al. (2022).

Transplantation of human iPSC-derived muscle stem cells in the diaphragm of

Duchenne muscular dystrophy model mice. PLoS One 17 (4), e0266391. doi:10.

1371/journal.pone.0266391

Cerletti, M., Jurga, S., Witczak, C. A., Hirshman, M. F., Shadrach, J. L., Goodyear, L. J.,

et al. (2008). Highly efficient, functional engraftment of skeletal muscle stem cells in

dystrophic muscles. Cell 134 (1), 37–47. doi:10.1016/j.cell.2008.05.049

Montarras, D., Morgan, J., Collins, C., Relaix, F., Zaffran, S., Cumano, A., et al. (2005).

Direct isolation of satellite cells for skeletal muscle regeneration. Science 309,

2064–2067.

Chamberlain, J. S., Metzger, J., Reyes, M., Townsend, D., Faulkner, J. A., Reyes, M.,

et al. (2007). Dystrophin deficient mdx mice display a reduced life span and are

susceptible to spontaneous rhabdomyosarcoma. FASEB J. 21 (9), 2195–2204.

Nakamura, A., and Takeda, S. (2011). Mammalian models of duchenne muscular

dystrophy: Pathological characteristics and therapeutic applications. J. Biomed.

Biotechnol. 2011, 184393. doi:10.1155/2011/184393

Danko, I., Chapman, V., and Wolff, J. A. (1992). The frequency of revertants in

mdx mouse genetic models for Duchenne muscular dystrophy. Pediatr. Res. 32,

128–131.

Nakamura, K., Fujii, W., Tsuboi, M., Tanihata, J., Teramoto, N., Takeuchi, S., et al.

(2014). Generation of muscular dystrophy model rats with a CRISPR/Cas system. Sci.

Rep. 4, 5635. doi:10.1038/srep05635

Doi, D., Magotani, H., Kikuchi, T., Ikeda, M., Hiramatsu, S., Yoshida, K., et al. (2020).

Pre-clinical study of induced pluripotent stem cell-derived dopaminergic progenitor

cells for Parkinson’s disease. Nat. Commun. 11 (1), 3369. doi:10.1038/s41467-02017165-w

Nalbandian, M., Zhao, M., Sasaki-Honda, M., Jonouchi, T., Lucena-Cacace, A.,

Mizusawa, T., et al. (2021). Characterization of hiPSC-derived muscle progenitors

reveals distinctive markers for myogenic cell purification toward cell therapy. Stem Cell

Rep. 16 (4), 883–898. doi:10.1016/j.stemcr.2021.03.004

Fairclough, R. J., Wood, M. J., and Davies, K. E. (2013). Therapy for duchenne

muscular dystrophy: Renewed optimism from genetic approaches. Nat. Rev. Genet. 14

(6), 373–378. doi:10.1038/nrg3460

Nowak, K. J., and Davies, K. E. (2004). Duchenne muscular dystrophy and

dystrophin: Pathogenesis and opportunities for treatment. EMBO Rep. 5 (9),

872–876. doi:10.1038/sj.embor.7400221

Goto, I., Peters, H. A., and Reese, H. H. (1967). Creatine phosphokinase in

neuromuscular disease. Patients and families. Activity Progressive Muscular

Dystrophy Neuromuscul. Dis. Aquatic Org. 16 (5), 529–535. doi:10.1001/archneur.

1967.00470230081011

Polavarapu, K., Manjunath, M., Preethish-Kumar, V., Sekar, D., Vengalil, S., Thomas,

P., et al. (2016). Muscle MRI in Duchenne muscular dystrophy: Evidence of a distinctive

pattern. Neuromuscul. Disord. 26 (11), 768–774. doi:10.1016/j.nmd.2016.09.002

Hanes, M. A. (2006). The nude rat American College of Laboratory Animal Medicine,

2nd Edition. The Laboratory Rat.

Roberts, R. G. (2001). Dystrophins and dystrobrevins. Genome Biol. 2 (4),

reviews3006.3001–3006.3007.

Hoffman, E. P., Brown, R. H., Jr., and Kunkel, L. M. (1987). Dystrophin: The protein

product of the duchenne muscular dystrophy locus. Cell 51, 919–928.

Sacco, A., Doyonnas, R., Kraft, P., Vitorovic, S., Blau, H. M., Kraft, P., et al. (2008).

Self-renewal and expansion of single transplanted muscle stem cells. Nature 456 (7221),

502–506. doi:10.1038/nature07384

Im, W. B., Phelps, S. F., Copen, E. H., Adams, E. G., Slightom, J. L., Chamberlain, J. S.,

et al. (1996). Differential expression of dystrophin isoforms in strains of mdx mice with

different mutations. Hum. Mol. Genet. 5 (8), 1149–1153.

Samata, B., Kikuchi, T., Miyawaki, Y., Morizane, A., Mashimo, T., Nakagawa, M., et al.

(2015). X-linked severe combined immunodeficiency (X-SCID) rats for xenotransplantation and behavioral evaluation. J. Neurosci. Methods 243, 68–77. doi:10.

1016/j.jneumeth.2015.01.027

Kornegay, J. N. (2017). The golden retriever model of Duchenne muscular dystrophy.

Skelet. Muscle 7 (1), 9. doi:10.1186/s13395-017-0124-z

Shiomi, K., Kiyono, T., Okamura, K., Uezumi, M., Goto, Y., Yasumoto, S., et al.

(2011). CDK4 and cyclin D1 allow human myogenic cells to recapture growth property

without compromising differentiation potential. Gene Ther. 18 (9), 857–866. doi:10.

1038/gt.2011.44

Larcher, T., Lafoux, A., Tesson, L., Remy, S., Thepenier, V., Francois, V., et al. (2014).

Characterization of dystrophin deficient rats: A new model for duchenne muscular

dystrophy. PLoS One 9 (10), e110371. doi:10.1371/journal.pone.0110371

Frontiers in Physiology

12

frontiersin.org

Sato et al.

10.3389/fphys.2023.1094359

Tanaka, K. K., Hall, J. K., Troy, A. A., Cornelison, D. D., Majka, S. M., Olwin, B. B.,

et al. (2009). Syndecan-4-expressing muscle progenitor cells in the SP engraft as satellite

cells during muscle regeneration. Cell Stem Cell 4 (3), 217–225. doi:10.1016/j.stem.2009.

01.016

Spurney, C. F., Gordish-Dressman, H., Guerron, A. D., Sali, A., Pandey, G. S., Rawat,

R., et al. (2009). Preclinical drug trials in the mdx mouse: Assessment of reliable and

sensitive outcome measures. Muscle Nerve 39 (5), 591–602. doi:10.1002/mus.21211

Sun, C., Serra, C., Lee, G., Wagner, R. K., Lee, G., and Wagner, K. R. (2020). Stem cellbased therapies for Duchenne muscular dystrophy. Exp. Neurol. 323, 113086. doi:10.

1016/j.expneurol.2019.113086

Veenvliet, J. V. B., Adriano, K., Helene, H., Leah, S-W., Manuela, S., Dennis, K., et al.

(2020). Mouse embryonic stem cells self-organize into trunklike structures with neural

tube and somites. Science 370, eaba4937. doi:10.1101/2020.03.04.974949

Taglietti, V., Kefi, K., Bronisz-Budzynska, I., Mirciloglu, B., Rodrigues, M., Cardone,

N., et al. (2022). Duchenne muscular dystrophy trajectory in R-DMDdel52 preclinical

rat model identifies COMP as biomarker of fibrosis. Acta Neuropathol. Commun. 10 (1),

60. doi:10.1186/s40478-022-01355-2

Vlahovic, H., Bazdaric, K., Marijancic, V., Soic-Vranic, T., Malnar, D., Arbanas, J.,

et al. (2017). Segmental fibre type composition of the rat iliopsoas muscle. J. Anat. 230

(4), 542–548. doi:10.1111/joa.12588

Xu, X., Wilschut, K. J., Kouklis, G., Tian, H., Hesse, R., Garland, C., et al. (2015).

Human satellite cell transplantation and regeneration from diverse skeletal muscles.

Stem Cell Rep. 5 (3), 419–434. doi:10.1016/j.stemcr.2015.07.016

Takenaka-Ninagawa, N., Kim, J., Zhao, M., Sato, M., Jonouchi, T., Goto, M., et al.

(2021). Collagen-VI supplementation by cell transplantation improves muscle

regeneration in Ullrich congenital muscular dystrophy model mice. Stem Cell Res.

Ther. 12 (1), 446. doi:10.1186/s13287-021-02514-3

Zhao, M., Tazumi, A., Takayama, S., Takenaka-Ninagawa, N., Nalbandian, M., Nagai,

M., et al. (2020). Induced fetal human muscle stem cells with high therapeutic potential

in a mouse muscular dystrophy model. Stem Cell Rep. 15 (1), 80–94. doi:10.1016/j.

stemcr.2020.06.004

Tanabe, Y., Esaki, K., Nomura, T., and Nomura, T. (1986). Skeletal muscle pathology

in X chromosome-linked muscular dystrophy (mdx) mouse. Acta Neuropathol. (Bet1)

69, 91–95.

Frontiers in Physiology

13

frontiersin.org

...

参考文献をもっと見る

全国の大学の
卒論・修論・学位論文

一発検索!

この論文の関連論文を見る