Results 91 to 100 of about 39,769 (244)
We propose a context‐dependent model where the Duchenne muscular dystrophy (DMD) gene acts as a tumour suppressor in aggressive tumours and as an oncogene in less aggressive ones. We propose this model as a unified framework to explain the opposing survival associations with DMD expression and to guide experimental exploration of the dual role of DMD ...
Lee Machado +4 more
wiley +1 more source
Perindopril preserves left ventricular function in X-linked Duchenne muscular dystrophy [PDF]
Denis Duboc +8 more
openalex +1 more source
Versatile Cell Penetrating Peptide for Multimodal CRISPR Gene Editing in Primary Stem Cells
CRISPR machinery in diverse molecular formats (DNA, RNA, and ribonucleic protein) is complexed into nanoparticles with the cell‐friendly arginine‐alanine‐leucine‐alanine (RALA) cell‐penetrating peptide. Nanoparticles are delivered to primary mesenchymal stem cells ex vivo or locally in vivo to facilitate multimodal CRISPR gene editing. This RALA‐CRISPR
Joshua P. Graham +9 more
wiley +1 more source
Impaired anti‐tumor immunity is mediated by TREM2 + macrophages in non‐small cell lung cancer. TREM2 + macrophages prevent anti‐tumor immunity depending on the limitation of CD4 + T and NK cells. TREM2 promotes cholesterol efflux via ABCA1 to limit the production of CX3CL1 in macrophages.
Yunhan Wang +9 more
wiley +1 more source
The guideline "Diagnosis and management of Duchenne muscular dystrophy" was supported by a 3-year-long project guided by US Centers for Disease Control and Prevention (CDC), in collaboration with patient advocacy groups [Muscular Dystrophy Association ...
Xi-hua LI
doaj
Introduction: Duchenne muscular dystrophy is a genetic X-linked recessive disorder. This condition is characterized by progressive loss of muscle tissue. Thus, it results in deterioration and inability to perform basic motor skills such as independent
Iwona Welian-Polus +9 more
doaj +1 more source
830. Highly Efficient and Comprehensive Gene Transfer to All Muscle Groups Necessary for the Potential Treatment of Duchenne/Becker Muscular Dystrophies Following In Utero Delivery of a Lentivirus Vector in Immuno-Competent Mice [PDF]
openalex +1 more source
Polymeric carriers provide versatile platforms for nucleic acid delivery. This review introduces a novel perspective inspired by protein structural organization to explore structure–function relationships of polymeric carriers, highlighting requirements of different nucleic acid payloads.
Hanieh Moradian +3 more
wiley +1 more source
Efficacy and safety of Viltepso® in Duchenne muscular dystrophy: review of clinical studies
The author presents a literature review on the safety and efficacy of antisense oligonucleotides in the treatment of Duchenne muscular dystrophy using the exon skipping method using Viltepso® (viltolarsen), the only drug of this class registered in ...
V. M. Suslov, D. I. Rudenko
doaj +1 more source
Combining genetics, neuropsychology and neuroimaging to improve understanding of brain involvement in Duchenne muscular dystrophy - a narrative review [PDF]
N. Doorenweerd
openalex +1 more source

