Rapid restitution of contractile dysfunction by synthetic copolymers in dystrophin-deficient single live skeletal muscle fibers

Duan D, Goemans N, Takeda S, Mercuri E, Aartsma-Rus A. Duchenne muscular dystrophy. Nat Rev Dis Primers. 2021;7(1):13.

Article  PubMed  Google Scholar 

Aartsma-Rus A, Van Deutekom JC, Fokkema IF, Van Ommen GJ, Den Dunnen JT. Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule. Muscle Nerve. 2006;34(2):135–44.

Article  CAS  PubMed  Google Scholar 

Moser H. Duchenne muscular dystrophy: pathogenetic aspects and genetic prevention. Hum Genet. 1984;66(1):17–40.

Article  CAS  PubMed  Google Scholar 

Yeung EW, Allen DG. Stretch-activated channels in stretch-induced muscle damage: role in muscular dystrophy. Clin Exp Pharmacol Physiol. 2004;31(8):551–6.

Article  CAS  PubMed  Google Scholar 

Hughes MC, Ramos SV, Turnbull PC, Rebalka IA, Cao A, Monaco CMF, et al. Early myopathy in Duchenne muscular dystrophy is associated with elevated mitochondrial H2 O2 emission during impaired oxidative phosphorylation. J Cachexia Sarcopenia Muscle. 2019;10(3):643–61.

Yanay N, Rabie M, Nevo Y. Impaired regeneration in dystrophic muscle-new target for therapy. Front Mol Neurosci. 2020;13:69.

CAS  PubMed  PubMed Central  Google Scholar 

Turner PR, Westwood T, Regen CM, Steinhardt RA. Increased protein degradation results from elevated free calcium levels found in muscle from mdx mice. Nature. 1988;335(6192):735–8.

Article  CAS  PubMed  Google Scholar 

Mareedu S, Million ED, Duan D, Babu GJ. Abnormal calcium handling in Duchenne muscular dystrophy: mechanisms and potential therapies. Front Physiol. 2021;12:647010.

Article  PubMed  PubMed Central  Google Scholar 

Garcia-Castaneda M, Michelucci A, Zhao N, Malik S, Dirksen RT. Postdevelopmental knockout of Orai1 improves muscle pathology in a mouse model of Duchenne muscular dystrophy. J Gen Physiol. 2022;154(9):e202213081.

Durbeej M, Campbell KP. Muscular dystrophies involving the dystrophin-glycoprotein complex: an overview of current mouse models. Curr Opin Genet Dev. 2002;12(3):349–61.

Article  CAS  PubMed  Google Scholar 

Hsu JD, Furumasu J. Gait and posture changes in the Duchenne muscular dystrophy child. Clin Orthop Relat Res. 1993;288:122–5.

Article  Google Scholar 

Lawler JM. Exacerbation of pathology by oxidative stress in respiratory and locomotor muscles with Duchenne muscular dystrophy. J Physiol. 2011;589(Pt 9):2161–70.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Boland BJ, Silbert PL, Groover RV, Wollan PC, Silverstein MD. Skeletal, cardiac, and smooth muscle failure in Duchenne muscular dystrophy. Pediatr Neurol. 1996;14(1):7–12.

Article  CAS  PubMed  Google Scholar 

De Ridder W, Nelson I, Asselbergh B, De Paepe B, Beuvin M, Ben Yaou R, et al. Muscular dystrophy with arrhythmia caused by loss-of-function mutations in BVES. Neurol Genet. 2019;5(2):e321.

Article  PubMed  PubMed Central  Google Scholar 

Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol. 2010;9(1):77–93.

Article  PubMed  Google Scholar 

Matthews E, Brassington R, Kuntzer T, Jichi F, Manzur AY. Corticosteroids for the treatment of Duchenne muscular dystrophy. Cochrane Database Syst Rev. 2016;5:CD003725.

Google Scholar 

Miyatake S, Shimizu-Motohashi Y, Takeda S, Aoki Y. Anti-inflammatory drugs for Duchenne muscular dystrophy: focus on skeletal muscle-releasing factors. Drug Des Devel Ther. 2016;10:2745–58.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Elangkovan N, Dickson G. Gene therapy for duchenne muscular dystrophy. J Neuromuscul Dis. 2021;8(s2):S303–16.

Article  PubMed  PubMed Central  Google Scholar 

Duan D. Micro-dystrophin gene therapy goes systemic in duchenne muscular dystrophy patients. Hum Gene Ther. 2018;29(7):733–6.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Crudele JM, Chamberlain JS. AAV-based gene therapies for the muscular dystrophies. Hum Mol Genet. 2019;28(R1):R102–7.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Pradhan S, Das A, Singh H, Chaturvedi S, Singh K, Mishra R, et al. Steroid responsiveness in Duchenne muscular dystrophy - predictive value of epigenetic regulator histone deacetylase 2. Indian J Pediatr. 2020;87(9):692–8.

Article  PubMed  Google Scholar 

Suthar R, Sahu JK. The Quest for the Prediction of Steroid Responsiveness in Duchenne Muscular Dystrophy. Indian J Pediatr. 2020;87(9):682–3.

Article  PubMed  Google Scholar 

Townsend D, Yasuda S, Chamberlain J, Metzger JM. Cardiac consequences to skeletal muscle-centric therapeutics for Duchenne muscular dystrophy. Trends Cardiovasc Med. 2009;19(2):50–5.

Article  CAS  PubMed  Google Scholar 

Houang EM, Haman KJ, Filareto A, Perlingeiro RC, Bates FS, Lowe DA, et al. Membrane-stabilizing copolymers confer marked protection to dystrophic skeletal muscle in vivo. Mol Ther Methods Clin Dev. 2015;2:15042.

Article  PubMed  PubMed Central  Google Scholar 

Houang EM, Haman KJ, Kim M, Zhang W, Lowe DA, Sham YY, et al. Chemical end group modified diblock copolymers elucidate anchor and chain mechanism of membrane stabilization. Mol Pharm. 2017;14(7):2333–9.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Marks JD, Pan CY, Bushell T, Cromie W, Lee RC. Amphiphilic, tri-block copolymers provide potent membrane-targeted neuroprotection. FASEB J. 2001;15(6):1107–9.

CAS  PubMed  Google Scholar 

Lee RC, River LP, Pan FS, Ji L, Wollmann RL. Surfactant-induced sealing of electropermeabilized skeletal muscle membranes in vivo. Proc Natl Acad Sci U S A. 1992;89(10):4524–8.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Lee RC, Hannig J, Matthews KL, Myerov A, Chen CT. Pharmaceutical therapies for sealing of permeabilized cell membranes in electrical injuries. Ann N Y Acad Sci. 1999;888:266–73.

Article  CAS  PubMed  Google Scholar 

Collins JM, Despa F, Lee RC. Structural and functional recovery of electropermeabilized skeletal muscle in-vivo after treatment with surfactant poloxamer 188. Biochim Biophys Acta. 2007;1768(5):1238–46.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Hunter RL, Luo AZ, Zhang R, Kozar RA, Moore FA. Poloxamer 188 inhibition of ischemia/reperfusion injury: evidence for a novel anti-adhesive mechanism. Ann Clin Lab Sci. 2010;40(2):115–25.

CAS  PubMed  Google Scholar 

Hannig J, Yu J, Beckett M, Weichselbaum R, Lee RC. Poloxamine 1107 sealing of radiopermeabilized erythrocyte membranes. Int J Radiat Biol. 1999;75(3):379–85.

Article  CAS  PubMed  Google Scholar 

Merchant FA, Holmes WH, Capelli-Schellpfeffer M, Lee RC, Toner M. Poloxamer 188 enhances functional recovery of lethally heat-shocked fibroblasts. J Surg Res. 1998;74(2):131–40.

Article  CAS  PubMed  Google Scholar 

Adams-Graves P, Kedar A, Koshy M, Steinberg M, Veith R, Ward D, et al. RheothRx (Poloxamer 188) injection for the acute painful episode of sickle cell disease: a pilot study. Blood. 1997;90(5):2041–6.

Article  CAS  PubMed  Google Scholar 

Ballas SK, Files B, Luchtman-Jones L, Benjamin L, Swerdlow P, Hilliard L, et al. Safety of purified poloxamer 188 in sickle cell disease: phase I study of a non-ionic surfactant in the management of acute chest syndrome. Hemoglobin. 2004;28(2):85–102.

Article  CAS  PubMed  Google Scholar 

Maskarinec SA, Hannig J, Lee RC, Lee KY. Direct observation of poloxamer 188 insertion into lipid monolayers. Biophys J. 2002;82(3):1453–9.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Yasuda S, Townsend D, Michele DE, Favre EG, Day SM, Metzger JM. Dystrophic heart failure blocked by membrane sealant poloxamer. Nature. 2005;436(7053):1025–9.

Article  CAS  PubMed  Google Scholar 

Houang EM, Sham YY, Bates FS, Metzger JM. Muscle membrane integrity in Duchenne muscular dystrophy: recent advances in copolymer-based muscle membrane stabilizers. Skelet Muscle. 2018;8(1):31.

Article  PubMed  PubMed Central  Google Scholar 

Bulfield G, Siller WG, Wight PA, Moore KJ. X chromosome-linked muscular dystrophy (mdx) in the mouse. Proc Natl Acad Sci USA. 1984;81(4):1189–92.

Article  CAS  PubMed  PubMed Central  Google Scholar 

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