Results 41 to 50 of about 28,350 (246)

Multi‐Omics Insights Into the Mechanisms of Early Muscle Fiber Difference and Transformation Between Lean‐Type and Chinese Indigenous Pigs

open access: yesAdvanced Science, EarlyView.
Multi‐omics analyses uncover breed‐specific cis‐regulatory landscapes and higher‐order chromatin architectural differences that underlie early postnatal muscle fiber divergence in pigs. A super‐enhancer upstream of PPP3CB recruits MEF2C to activate PPP3CB transcription, while the PPP3CB–MEF2C positive feedback loop promotes oxidative muscle fiber ...
Shuailong Zheng   +8 more
wiley   +1 more source

An investigation into the effects of dystrophin on the lateral mobility of muscle membrane components. [PDF]

open access: yes, 1999
Dystrophin is the product of the Duchenne Muscular Dystrophy gene locus, whose absence results in progressive skeletal muscle breakdown. Despite considerable work on the localisation of dystrophin and its associated complex, its role in muscle function ...
Dutton, A.L., Dutton, Anna Louise
core  

Effects of Mini-Dystrophin on Dystrophin-Deficient, Human Skeletal Muscle-Derived Cells [PDF]

open access: yes, 2020
Background: We are developing a novel therapy for Duchenne muscular dystrophy (DMD), involving the transplantation of autologous, skeletal muscle-derived stem cells that have been genetically corrected to express dystrophin.
Jennifer E. Morgan   +2 more
core   +2 more sources

Systemic delivery of full-length dystrophin in Duchenne muscular dystrophy mice

open access: yesNature Communications
Current 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 (
Yuan Zhou   +4 more
doaj   +1 more source

Myelination is delayed during postnatal brain development in the mdx mouse model of Duchenne muscular dystrophy

open access: yesBMC Neuroscience, 2017
Background In Duchenne muscular dystrophy (DMD), the loss of the dystrophin component of the dystrophin-glycoprotein complex (DGC) compromises plasma membrane integrity in skeletal muscle, resulting in extensive muscle degeneration. In addition, many DMD
Azeez Aranmolate   +2 more
doaj   +1 more source

Promising therapeutic approaches of utrophin replacing dystrophin in the treatment of Duchenne muscular dystrophy

open access: yesFundamental Research, 2022
Duchenne muscular dystrophy (DMD) is a serious genetic neuromuscular rare disease that is prevalent and caused by the mutation/deletion of the X-linked DMD gene that encodes dystrophin.
Ruo Wu   +3 more
doaj   +1 more source

Endogenous Engineering Reprograms Extracellular Vesicles for Enhanced Therapeutic Function

open access: yesAdvanced Science, EarlyView.
This review explains how Extracellular vesicles‐producing cells can be endogenously engineered to load therapeutic proteins and nucleic acids. We summarize physiological and genetic strategies that harness native sorting pathways for selective cargo loading.
Jinghui Wang   +10 more
wiley   +1 more source

Dystrophin Dp71 Subisoforms Localize to the Mitochondria of Human Cells

open access: yesLife, 2021
Duchenne muscular dystrophy (DMD) is a fatal muscle wasting disease caused by deficiency in dystrophin, a protein product encoded by the DMD gene. Mitochondrial dysfunction is now attracting much attention as a central player in DMD pathology.
Emma Tabe Eko Niba   +6 more
doaj   +1 more source

Investigating the Role of Type I Interferon Signaling on Muscle Disease Using Mouse Models

open access: yesArthritis &Rheumatology, EarlyView.
Objective Dysregulated type I interferon (IFN) signaling contributes to autoimmune myositis pathogenesis. We investigated the therapeutic effects of JAK inhibitors in two mouse models. We also examined how type I IFNs affect muscle vasculature. Methods Myositis was induced in major histocompatibility complex class I double transgenic ([TRE‐H‐2Kb (H ...
Rita Spathis   +11 more
wiley   +1 more source

Status and future of recombinant adeno‐associated virus vector manufacturing

open access: yesBiotechnology Progress, EarlyView.
Abstract Sixty years of adeno‐associated virus (AAV) research illustrates a trajectory marked by basic science exploration, iterative innovation, persistent challenges, a number of clinical setbacks, as well as commercial therapeutic triumphs. This continual evolution has led to recombinant AAV (rAAV) becoming a cornerstone of modern gene therapy ...
Frank Agbogbo, David Dismuke
wiley   +1 more source

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