One-Dimensional Nickel Molybdate Nanostructures with Enhanced Supercapacitor Performance.

One-dimensional NiMoO nanofibers were successfully prepared by electrospinning and high-temperature calcination. The supercapacitor performance tests were conducted on the prepared materials in a three-electrode system, and it was found that the calcination temperature during the preparation of the fibers seriously affects the final morphology and electrochemical performance of the obtained samples. The sample with a calcination temperature of 500 °C has better performance, its specific capacitance can reach 1947 F g, and the retention rate is 82.

Gene therapy for glycogen storage diseases.

Glycogen storage disorders (GSDs) are inherited disorders of metabolism resulting from the deficiency of individual enzymes involved in the synthesis, transport, and degradation of glycogen. This literature review summarizes the development of gene therapy for the GSDs. The abnormal accumulation of glycogen and deficiency of glucose production in GSDs lead to unique symptoms based upon the enzyme step and tissues involved, such as liver and kidney involvement associated with severe hypoglycemia during fasting and the risk of long-term complications including hepatic adenoma/carcinoma and end stage kidney disease in GSD Ia from glucose-6-phosphatase deficiency, and cardiac/skeletal/smooth muscle involvement associated with myopathy +/- cardiomyopathy and the risk for cardiorespiratory failure in Pompe disease.

Successful AAV8 readministration: Suppression of capsid-specific neutralizing antibodies by a combination treatment of bortezomib and CD20 mAb in a mouse model of Pompe disease.

Background: A major challenge to adeno-associated virus (AAV)-mediated gene therapy is the presence of anti-AAV capsid neutralizing antibodies (NAbs), which can block viral vector transduction even at very low titers. In the present study, we examined the ability of a combination immunosuppression (IS) treatment with bortezomib and a mouse-specific CD20 monoclonal antibody to suppress anti-AAV NAbs and enable readministration of AAV vectors of the same capsid in mice.

Methods: An AAV8 vector (AAV8-CB-hGAA) that ubiquitously expresses human α-glucosidase was used for initial gene therapy and a second AAV8 vector (AAV8-LSP-hSEAP) that contains a liver-specific promoter to express human secreted embryonic alkaline phosphatase (hSEAP) was used for AAV readministration.

Clinical significance of anti-rheumatoid arthritis 33 antibody in patients with systemic lupus erythematosus.

Although anti-rheumatoid arthritis (RA) 33 antibodies have been reported to be present in various connective tissue diseases (CTDs), the clinical significance of anti-RA33 in CTDs is still obscure. This study was performed to explore the clinical significance of anti-RA33 in CTDs, especially systemic lupus erythematosus (SLE). A total of 565 patients with positive anti-nuclear antibodies who had been tested for anti-RA33 were included in this study and were further classified into RA33-positive and RA33-negative groups.

Suppression of pullulanase-induced cytotoxic T cell response with a dual promoter in GSD IIIa mice.

Glycogen debranching enzyme deficiency in glycogen storage disease type III (GSD III) results in excessive glycogen accumulation in multiple tissues, primarily the liver, heart, and skeletal muscle. We recently reported that an adeno-associated virus vector expressing a bacterial debranching enzyme (pullulanase) driven by the ubiquitous CMV enhancer/chicken β-actin (CB) promoter cleared glycogen in major affected tissues of infant GSD IIIa mice. In this study, we developed a potentially novel dual promoter consisting of a liver-specific promoter (LSP) and the CB promoter for gene therapy in adult GSD IIIa mice.

Evaluation of carbon neutrality capacity based on a novel comprehensive model.

The Chinese government actively participates in global climate governance and has proposed to achieve the goal of carbon neutrality by 2060. Due to large differences in regional development, local governments need to comprehend their own carbon neutrality status and then scientifically plan a path to achieve carbon neutrality. In this study, we constructed a new carbon neutrality capacity evaluation indicator system named CNCIS, which can dynamically reflect the balance of energy, economy and environment in the process of reducing carbon emissions.

Boronate-modified polyethyleneimine dendrimer as a solid-phase extraction adsorbent for the analysis of luteolin HPLC.

Luteolin (LTL) is a flavonoid containing a -diol, which has significant anti-inflammatory, anti-allergic, anti-diabetic, anti-cancer and neuroprotective activities. In this work, a silver modified boric acid affinity polyvinyl imine (PEI) dendritic adsorbent (PPEI-Ag@CPBA) was prepared on polystyrene (PS) for the rapid recognition and selective separation of LTL. A thin layer of polydopamine (PDA) was formed on the surface of the substrate by self-polymerization, and a PDA-coated PS material (PS@PDA) was obtained.

Case Report: IgA Nephropathy in a Patient With Anti-Transcription Intermediary Factor-1γ Antibody-Positive Dermatomyositis.

Immunoglobulin A nephropathy (IgAN) is the most common primary glomerulonephritis characterized by IgA deposits in the mesangial area of glomeruli. Connective tissue disorders are some of the most frequent causes of secondary IgAN. Nevertheless, IgAN rarely occurs in systemic autoimmune myopathies (SAMs).

Metal oxide-based macroporous ordered double affinity molecularly imprinted polymer for specific separation and enrichment of glycoprotein from food samples: a co-modification of DMSA and boronate affinity.

Metal oxide-based macroporous ordered double affinity molecularly imprinted polymers (D-MIPs) were developed as solid phase extraction (SPE) adsorbents for the specific identification of ovalbumin (OVA) under physiological pH conditions prior to ultraviolet visible (UV-vis) spectrophotometric detection. Herein, macroporous alumina (MA) was used as a matrix; dimercaptosuccinic acid (DMSA) and 3-aminophenylboric acid (APBA) were employed as dual-functional monomers; APBA is a self-polymerizing monomer. The effects of synthesis conditions, SPE conditions as well as selectivity, reproducibility, and reusability were studied.

New Insights into Gastrointestinal Involvement in Late-Onset Pompe Disease: Lessons Learned from Bench and Bedside.

Background: There are new emerging phenotypes in Pompe disease, and studies on smooth muscle pathology are limited. Gastrointestinal (GI) manifestations are poorly understood and underreported in Pompe disease.

Methods: To understand the extent and the effects of enzyme replacement therapy (ERT; alglucosidase alfa) in Pompe disease, we studied the histopathology (entire GI tract) in Pompe mice (GAAKO 6/6).

Characterization of liver GSD IX γ2 pathophysiology in a novel Phkg2 mouse model.

Introduction: Liver Glycogen Storage Disease IX is a rare metabolic disorder of glycogen metabolism caused by deficiency of the phosphorylase kinase enzyme (PhK). Variants in the PHKG2 gene, encoding the liver-specific catalytic γ2 subunit of PhK, are associated with a liver GSD IX subtype known as PHKG2 GSD IX or GSD IX γ2. There is emerging evidence that patients with GSD IX γ2 can develop severe and progressive liver disease, yet research regarding the disease has been minimal to date.

Elevated interleukin-37 associated with tophus and pro-inflammatory mediators in Chinese gout patients.

Introduction: Interleukin-37(IL-37), a natural inhibitor of innate immunity, has been identified to protect against various inflammatory diseases, including monosodium urate (MSU)-induced inflammation. However, the association of IL-37 with clinical indexes and pro-inflammatory mediators in gout patients remains unclear. The aim of this study was to determine IL-37 level in hyperuricemia and gout patients with or without tophus, and to investigate the correlations of IL-37 with clinical indexs such as Uric Acid (UA), CRP(C-reactive protein), Creatinine Clearance Rate (Ccr), Erythrocyte Sedimentation Rate (ESR) and so on, as well as with the pro-inflammatory mediators in serum including Interleukin-1β(IL-1β), Interleukin-6(IL-6) and Interleukin-18(IL-18) from gout patients.

Transcriptomic and Proteomic Analysis of Clear Cell Foci (CCF) in the Human Non-Cirrhotic Liver Identifies Several Differentially Expressed Genes and Proteins with Functions in Cancer Cell Biology and Glycogen Metabolism.

Clear cell foci (CCF) of the liver are considered to be pre-neoplastic lesions of hepatocellular adenomas and carcinomas. They are hallmarked by glycogen overload and activation of AKT (v-akt murine thymoma viral oncogene homolog)/mTOR (mammalian target of rapamycin)-signaling. Here, we report the transcriptome and proteome of CCF extracted from human liver biopsies by laser capture microdissection.

A Novel Gene Therapy Approach for GSD III Using an AAV Vector Encoding a Bacterial Glycogen Debranching Enzyme.

Glycogen storage disease type III (GSD III) is an inherited disorder caused by a deficiency of glycogen debranching enzyme (GDE), which results in the accumulation of abnormal glycogen (limit dextrin) in the cytoplasm of liver, heart, and skeletal muscle cells. Currently, there is no curative treatment for this disease. Gene therapy with adeno-associated virus (AAV) provides an optimal treatment approach for monogenic diseases like GSD III.

[Effect of acupuncture combined with repetitive transcranial magnetic stimulation on motor function and cerebral hemodynamics in children with spastic cerebral palsy with spleen-kidney deficiency].

Objective: To investigate the clinical effect of acupuncture combined with repetitive transcranial magnetic stimulation in the treatment of children with spastic cerebral palsy with spleen-kidney deficiency, as well as its effect in improving cerebral hemodynamics.

Methods: A total of 220 children with spastic cerebral palsy were divided into observation group and control group using a random number table, with 110 children in each group. The children in the control group were given rehabilitation training and repetitive transcranial magnetic stimulation, and those in the observation group were given acupuncture in addition to the treatment in the control group.

An emerging phenotype of central nervous system involvement in Pompe disease: from bench to bedside and beyond.

Pompe disease (PD) is a lysosomal storage disorder caused by deficiency of the lysosomal enzyme acid-alpha glucosidase (GAA). Pathogenic variants in the GAA gene lead to excessive accumulation of lysosomal glycogen primarily in the cardiac, skeletal, and smooth muscles. There is growing evidence of central nervous system (CNS) involvement in PD.

Gene therapy for glycogen storage diseases.

The focus of this review is the development of gene therapy for glycogen storage diseases (GSDs). GSD results from the deficiency of specific enzymes involved in the storage and retrieval of glucose in the body. Broadly, GSDs can be divided into types that affect liver or muscle or both tissues.

Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease.

Pompe disease, a severe and often fatal neuromuscular disorder, is caused by a deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA). The disease is characterized by the accumulation of excess glycogen in the heart, skeletal muscle, and CNS. Currently approved enzyme replacement therapy or experimental adeno-associated virus (AAV)-mediated gene therapy has little effect on CNS correction.

Therapeutic Benefit of Autophagy Modulation in Pompe Disease.

The complexity of the pathogenic cascade in lysosomal storage disorders suggests that combination therapy will be needed to target various aspects of pathogenesis. The standard of care for Pompe disease (glycogen storage disease type II), a deficiency of lysosomal acid alpha glucosidase, is enzyme replacement therapy (ERT). Many patients have poor outcomes due to limited efficacy of the drug in clearing muscle glycogen stores.

IL-37 is associated with osteoarthritis disease activity and suppresses proinflammatory cytokines production in synovial cells.

The objective of this study is to investigate the correlation between IL-37 level and osteoarthritis activity and to determine the anti-inflammatory effects of IL-37 in peripheral blood mononuclear cells (PBMCs) and synovial cells (SCs) from osteoarthritis (OA) patients, which including 32 patients with erosive inflammatory OA (EIOA) and 40 patients with primary generalized OA (PGOA), 40 age and sex matched healthy volunteers were recruited as healthy controls (HCs). The protein and relative mRNA levels of IL-37 were significant increased in the blood of EIOA patients compared with those of PGOA patients and HCs. Serum IL-37 levels of OA patients were positively correlated with VAS score, as well as with CRP, ESR in blood.

A pilot study on using rapamycin-carrying synthetic vaccine particles (SVP) in conjunction with enzyme replacement therapy to induce immune tolerance in Pompe disease.

A major obstacle to enzyme replacement therapy (ERT) with recombinant human acid-α-glucosidase (rhGAA) for Pompe disease is the development of high titers of anti-rhGAA antibodies in a subset of patients, which often leads to a loss of treatment efficacy. In an effort to induce sustained immune tolerance to rhGAA, we supplemented the rhGAA therapy with a weekly intravenous injection of synthetic vaccine particles carrying rapamycin (SVP-Rapa) during the first 3 weeks of a 12-week course of ERT in GAA-KO mice, and compared this with three intraperitoneal injections of methotrexate (MTX) per week for the first 3 weeks. Empty nanoparticles (NP) were used as negative control for SVP-Rapa.

Antibody-mediated enzyme replacement therapy targeting both lysosomal and cytoplasmic glycogen in Pompe disease.

Unlabelled: Pompe disease is characterized by accumulation of both lysosomal and cytoplasmic glycogen primarily in skeletal and cardiac muscles. Mannose-6-phosphate receptor-mediated enzyme replacement therapy (ERT) with recombinant human acid α-glucosidase (rhGAA) targets the enzyme to lysosomes and thus is unable to digest cytoplasmic glycogen. Studies have shown that anti-DNA antibody 3E10 penetrates living cells and delivers "cargo" proteins to the cytosol or nucleus via equilibrative nucleoside transporter ENT2.

Systemic Correction of Murine Glycogen Storage Disease Type IV by an AAV-Mediated Gene Therapy.

Deficiency of glycogen branching enzyme (GBE) causes glycogen storage disease type IV (GSD IV), which is characterized by the accumulation of a less branched, poorly soluble form of glycogen called polyglucosan (PG) in multiple tissues. This study evaluates the efficacy of gene therapy with an adeno-associated viral (AAV) vector in a mouse model of adult form of GSD IV (Gbe1). An AAV serotype 9 (AAV9) vector containing a human GBE expression cassette (AAV-GBE) was intravenously injected into 14-day-old Gbe1 mice at a dose of 5 × 10 vector genomes per mouse.