Browsing by Subject "Sarcolemma"
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Item Development and characterization of diffusion membrane potentials in canine cardiac sarcolemmal vesicles(1981) Maddock, Stephen WilliamItem Dynamic regulation of the subunit composition of BK channels in smooth muscle(American Heart Association, 2017-09-01) Dick, Gregory M.; Tune, Johnathan D.; Cellular and Integrative Physiology, School of MedicineItem In vivo manipulation of adenylate cyclase inhibition in canine cardiac sarcolemma(1988) Hodges, Timothy D.Item Lanthanum augmentation of ATP-dependent calcium accumulation into sarcolemmal vesicles(1988) Anderson, Thomas BedfordItem Systemic delivery of full-length dystrophin in Duchenne muscular dystrophy mice(Springer Nature, 2024-07-21) Zhou, Yuan; Zhang, Chen; Xiao, Weidong; Herzog, Roland W.; Han, Renzhi; Pediatrics, School of MedicineCurrent gene therapy for Duchenne muscular dystrophy (DMD) utilizes adeno-associated virus (AAV) to deliver micro-dystrophin (µDys), which does not provide full protection for striated muscles as it lacks many important functional domains of full-length (FL) dystrophin. Here we develop a triple vector system to deliver FL-dystrophin into skeletal and cardiac muscles. We split FL-dystrophin into three fragments linked to two orthogonal pairs of split intein, allowing efficient assembly of FL-dystrophin. The three fragments packaged in myotropic AAV (MyoAAV4A) restore FL-dystrophin expression in both skeletal and cardiac muscles in male mdx4cv mice. Dystrophin-glycoprotein complex components are also restored at the sarcolemma of dystrophic muscles. MyoAAV4A-delivered FL-dystrophin significantly improves muscle histopathology, contractility, and overall strength comparable to µDys, but unlike µDys, it also restores defective cavin 4 localization and associated signaling in mdx4cv heart. Therefore, our data support the feasibility of a mutation-independent FL-dystrophin gene therapy for DMD, warranting further clinical development.