Results 131 to 140 of about 39,875 (271)
Endogenous Engineering Reprograms Extracellular Vesicles for Enhanced Therapeutic Function
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
Duchenne Muscular Dystrophy [PDF]
Progressive muscular damage and degeneration occurs in people with Duchenne Muscular Dystrophy (DMD)
core +1 more source
Atrogin-1 promotes muscle homeostasis by regulating levels of endoplasmic reticulum chaperone BiP
Skeletal muscle wasting results from numerous pathological conditions affecting both the musculoskeletal and nervous systems. A unifying feature of these pathologies is the upregulation of members of the E3 ubiquitin ligase family, resulting in increased
Avnika A. Ruparelia +12 more
doaj +1 more source
A Mechanistic Model of rAAV Production in Synthetic Cell Lines
ABSTRACT The recombinant adeno‐associated virus (rAAV) is a widely used vector for gene therapy. Its manufacturing faces significant challenges in producing the large quantities of vectors needed for clinical applications and reducing empty particles.
Prahalad Srinivasan +5 more
wiley +1 more source
Computer task performance by subjects with Duchenne muscular dystrophy
Silvia Regina Pinheiro Malheiros,1 Talita Dias da Silva,2 Francis Meire Favero,2 Luiz Carlos de Abreu,1 Felipe Fregni,3 Denise Cardoso Ribeiro,4 Carlos Bandeira de Mello Monteiro1,4,5 1School of Medicine of ABC, Santo Andre, Brazil; 2Department of ...
Ribeiro DC +6 more
core
ABSTRACT Objective Pediatric neuropalliative medicine (PNPM) is a recently developed area of subspecialty neurology practice focused on supporting the complex emotional, psychological, and physical aspects of caring for a child with serious neurological disease.
Lauren Treat +2 more
wiley +1 more source
N‐acetyltransferase 10 (NAT10) catalyses N4‐acetylcytidine (ac4C) modification of PML pre‐mRNA, shifting splicing from the senescence‐promoting PML‐FL to the senescence‐inhibiting PML‐S isoform via SRSF1. This rejuvenates adipose‐derived stem cells (ADSCs) by reducing senescence markers and senescence‐associated secretory phenotype (SASP), thereby ...
Wuhan Wei +9 more
wiley +1 more source
Modification of the myostatin and nitric oxide signalling pathways as treatments for Duchenne Muscular Dystrophy [PDF]
Duchenne muscular dystrophy (DMD) is a fatal X-linked disease affecting 1 in 3500 live male births that arises from faulty production of the sub-sarcolemmal protein, dystrophin.
Marrett, Garth
core
1. Lactate‐derived histone lactylation links glycolytic reprogramming to epigenetic regulation in female reproductive diseases. 2. In endometriosis, lactylation may promote inflammatory remodelling, immune dysregulation and ferroptosis resistance. 3. In PCOS, lactylation connects metabolic disturbance to granulosa‐cell dysfunction and hyperandrogenism.
Jiajun Qiao +5 more
wiley +1 more source
Noncompaction in Duchenne Muscular Dystrophy
Finsterer, Josef, Zarrouk-Mahjoub, Sinda
openaire +6 more sources

