Loss-of-function mutations in SMN1 cause spinal muscular atrophy (SMA), a leading genetic cause of infant mortality. The related SMN2 gene expresses suboptimal levels of functional SMN protein, due to a splicing defect. Many SMA patients reach adulthood, and there is also adult-onset (type IV) SMA. There is currently no animal model for adult-onset SMA, and the tissue-specific pathogenesis of p...

Osteopontin (OPN) has an important role in hepatocellular carcinoma (HCC) progression and metastasis. This study was to investigate the therapeutic potential of inhibition of OPN expression. A 2'-O-methoxyethylribose-modified phosphorothioate antisense oligonucleotides (ASO) was used to knock-down OPN expression in the human metastatic HCC cell line HCCLM6 and in nude mice orthotopically implan...

The crystal structure of durangite, ideally NaAl(AsO(4))F (chemical name sodium aluminium arsenate fluoride), has been determined previously [Kokkoros (1938). Z. Kristallogr.99, 38-49] using Weissenberg film data without reporting displacement parameters of atoms or a reliability factor. This study reports the redetermination of the structure of durangite using single-crystal X-ray diffraction ...

OBJECTIVE SSc causes intractable ischaemic ulcers. To avoid major amputation, we examined the safety and efficacy of therapeutic vascular angiogenesis for digital ulcers due to SSc. METHODS A single-centre, open-label pilot study was conducted in patients with an ischaemic digital ulcer [n = 40, mean age 65 years (s.d. 8), Rutherford class III-5 or III-6) due to lcSSc (n = 11) or arterioscler...

Numerous studies have suggested that alpha-synuclein plays a prominent role in both familial and idiopathic Parkinson's disease (PD). Mice in which human alpha-synuclein is overexpressed (ASO) display progressive motor deficits and many nonmotor features of PD. However, it is unclear what in vivo pathophysiological mechanisms drive these motor deficits. It is also unknown whether previously pro...

An analysis of the relationship between changes in Arctic stratospheric ozone (ASO) and precipitation eastern North America (38°–54°N, 65°–87°W; PENA) was performed using observational reanalysis data coupled with Whole Atmosphere Community Climate Model version 4 (WACCM4). We found that March ASO exhibits a strong correlation PENA April, indicating one-month leading exerts potentially impact o...

The structure of the title salt adduct, (C(4)H(9))(4)N(+)·C(6)H(5)AsO(3)H(-)·C(6)H(5)AsO(3)H(2), features chains along the a axis comprising alternating hydrogen phenyl-arsonate anions and phenyl-arsonic acid mol-ecules linked by O-H⋯O hydrogen bonds.

BACKGROUND In adults with prior arterial switch operation (ASO) for d-transposition of the great arteries, the need for routine coronary artery assessment and evaluation for silent myocardial ischemia is not well defined. In this observational study we aimed to determine the value of a comprehensive cardiovascular magnetic resonance (CMR) protocol for the detection of coronary problems in adult...

BACKGROUND The proprotein convertase subtilisin/kexin type 9 (PCSK9) is an important factor in the etiology of familial hypercholesterolemia (FH) and is also an attractive therapeutic target to reduce low density lipoprotein (LDL) cholesterol. PCSK9 accelerates the degradation of hepatic low density lipoprotein receptor (LDLR) and low levels of hepatic PCSK9 activity are associated with reduced...

Tumor metastasis occurs naturally in pancreatic cancer, and the efficacy of chemotherapy is usually poor. Precision medicine, combining downregulation of target genes with chemotherapy drugs, is expected to improve therapeutic effects. Therefore, we developed a combined therapy of microRNA-21 antisense oligonucleotides (ASO-miR-21) and gemcitabine (Gem) using a targeted co-delivery nanoparticle...