Intravital imaging of muscle damage and response to therapy in a model of Pompe disease

Naresh K. Meena; Yeap Ng; Davide Randazzo; Roberto Weigert; Rosa Puertollano; Nina Raben

doi:10.1002/ctm2.1561

Clinical and Translational Medicine ›› 2024, Vol. 14 ›› Issue (3) : e1561 DOI: 10.1002/ctm2.1561

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Intravital imaging of muscle damage and response to therapy in a model of Pompe disease

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Naresh K. Meena, Yeap Ng, Davide Randazzo, Roberto Weigert, Rosa Puertollano, Nina Raben. Intravital imaging of muscle damage and response to therapy in a model of Pompe disease. Clinical and Translational Medicine, 2024, 14(3): e1561 DOI:10.1002/ctm2.1561

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References

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[1]	MeenaNK, RabenN. Pompe disease: new developments in an old lysosomal storage disorder. Biomolecules. 2020;10(9):1339.

[2]	MeenaNK, Randazzo D, RabenN, PuertollanoR. AAV-mediated delivery of secreted acid alpha-glucosidase with enhanced uptake corrects neuromuscular pathology in Pompe mice. JCI Insight. 2023;8(16):e170199.

[3]	MeenaNK, Ralston E, RabenN, PuertollanoR. Enzyme replacement therapy can reverse pathogenic cascade in Pompe disease. Mol Ther Methods Clin Dev. 2020;18:199-214.

[4]	XuS, LunY, FrascellaM, Garcia A, SoskaR, NairA, et al. Improved efficacy of a next-generation ERT in murine Pompe disease. JCI Insight. 2019;4(5):e125358.

[5]	KolencOI, QuinnKP. Evaluating Cell Metabolism Through Autofluorescence Imaging of NAD(P)H and FAD. Antioxid Redox Sign. 2019;30(6):875-889.

[6]	WeigertR, Porat-Shliom N, AmornphimolthamP. Imaging cell biology in live animals: ready for prime time. J Cell Biol. 2013;201(7):969-979.

[7]	Porat-ShliomN, ChenY, ToraM, Shitara A, MasedunskasA, WeigertR. In vivo tissue-wide synchronization of mitochondrial metabolic oscillations. Cell Rep. 2014;9(2):514-521. PMCPMC4223640.

[8]	LimJA, LiL, KakhlonO, Myerowitz R, RabenN. Defects in calcium homeostasis and mitochondria can be reversed in Pompe disease. Autophagy 2015;11(2):385-402.

[9]	TaralloA, Damiano C, StrolloS, et al. Correction of oxidative stress enhances enzyme replacement therapy in Pompe disease. EMBO Mol Med. 2021;13(11):e14434.

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Published 2024. This article is a U.S. Government work and is in the public domain in the USA. Clinical and Translational Medicine published by John Wiley & Sons Australia, Ltd on behalf of Shanghai Institute of Clinical Bioinformatics.