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
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