A cell-penetrating peptide (CPP)-morpholino oligonucleotide (MO) conjugate (PMO) that has an antibiotic effect in culture had some contaminating CPPs in earlier preparations. The mixed conjugate had gene-specific and gene-nonspecific effects. An improved purification procedure separates the PMO from the free CPP and MO. The gene-specific effects are a result of the PMO, and the nonspecific effe...

Carbohydrate-based infusion solutions are widely used in the clinic. Here we show that co-administration of phosphorodiamidate morpholino oligomers (PMOs) with glucose enhances exon-skipping activity in Duchenne muscular dystrophy (DMD) mdx mice. We identify a glucose-fructose (GF) formulation that potentiates PMO activity, completely corrects aberrant Dmd transcripts, restores dystrophin level...

There has been much speculation on the importance of emotional factors in patients with immune-mediated inflammatory disease (IMID); it is only in the past 10 years that well designed, large-cohort studies have been able to clarify this relationship. This article provides an overview of evidence on the occurrence of depression and anxiety in IMID, and the role of these comorbidities as risk fac...

BACKGROUND Antisense-mediated exon skipping is a putative treatment for Duchenne muscular dystrophy (DMD). Using antisense oligonucleotides (AONs), the disrupted DMD reading frame is restored, allowing generation of partially functional dystrophin and conversion of a severe Duchenne into a milder Becker muscular dystrophy phenotype. In vivo studies are mainly performed using 2'-O-methyl phospho...

Phosphorodiamidate morpholino oligomer (PMO)-mediated exon skipping is among the more promising approaches to the treatment of several neuromuscular disorders including Duchenne muscular dystrophy. The main weakness of this approach arises from the low efficiency and sporadic nature of the delivery of charge-neutral PMO into muscle fibers, the mechanism of which is unknown. In this study, to te...

BACKGROUND Postmenopausal osteoporosis (PMO) is an estrogen deficiency condition that causes severe loss of bone mass in the vertebrae and long bones. We explored the effect and the possible underlying mechanism of the extracts of Astragalus (AE) on the tooth alveolar bone rebuilding progress of postmenopausal osteoporosis of PMO animal models. MATERIAL AND METHODS The PMO models were acquire...

The excited state characteristics of phenylene (Ph)-bridged periodic mesoporous organosilica (PMO) powders with crystal-like and amorphous wall structures are investigated. Crystal-like Ph-PMO has a molecular ordering of the bridging organic moieties with intervals of 0.76 and 0.44 nm parallel and perpendicular to the mesochannel direction, respectively, whereas amorphous Ph-PMO has no molecula...

Phosphorodiamidate morpholino oligomers (PMO) inhibit targeted gene expression by preventing ribosomal assembly, thereby preventing mRNA translation. AVI-4126, a PMO targeted against c-MYC, has been extensively characterized in multiple cancer and other disease models and is currently in human clinical trials. A phase I clinical study was conducted to address the issue of PMO bioavailability in...

The current study assessed the acute effects of pacemaker optimization (PMO) on cardiac function using echocardiographic (ECHO) tissue Doppler imaging (TDI) in the post CRT setting. Data were analyzed from 50 consecutive patients clinically referred for PMO. Patients underwent a sequential ECHO/TDIguided PMO study to determine optimal pacemaker settings. In 34 of 50 patients a change in pacemak...

A contemporary question in the intensely active field of periodic mesoporous organosilica (PMO) materials is how large a silsesquioxane precursor can be self-assembled under template direction into the pore walls of an ordered mesostructure. An answer to this question is beginning to emerge with the ability to synthesize dendrimer, buckyball, and polyhedral oligomeric silsesquioxane PMOs. In th...