Longitudinal imaging of therapeutic enzyme expression after gene therapy for Fabry disease using positron emission tomography and the radiotracer [F]AGAL.

Longitudinal, non-invasive, in vivo monitoring of therapeutic gene expression is an unmet need for gene therapy (GT). Positron emission tomography (PET) radiotracers designed to bind to therapeutic proteins may provide a sensitive imaging platform to guide treatment response and dose optimization in GT. Herein, we evaluate a novel PET tracer ([F]AGAL) for targeting α-galactosidase A (GLA), an enzyme deficient in Fabry disease.

A novel ZnO-TiO-BiWO/Carboxymethyl chitosan composite with high antimicrobial activity and visible-light catalytic degradation of ethylene towards banana preservation in hot and humid environments.

In order to solve the problem of significantly shortened storage time of bananas in hot and humid environment (e.g., 30 °C and 90 % relative humidity), this paper reports the preparation of ZnO-TiO-BiWO (ZTB) ternary heterojunction antimicrobial photocatalysts composite carboxymethyl chitosan film (ZTB/CMCS).

A Potential Neurotoxic Mechanism: Bisphenol S-Induced Inhibition of Glucose Transporter 1 Leads to ATP Excitotoxicity in the Zebrafish Brain.

Many environmental pollutants have neurotoxic effects, but the initial molecular events involved in these effects are unclear. Here, zebrafish were exposed to the neurotoxicant bisphenol S (BPS, 1, 10, or 100 μg/L) from the embryonic stage to the larval stage to explore the ability of BPS to interfere with energy metabolism in the brain. BPS, which is similar to a glucose transporter 1 (GLUT1) inhibitor, inhibited GLUT1 function but increased mitochondrial activity in the brains of larval zebrafish.

Developing Gene Therapy for Mitigating Multisystemic Pathology in Fabry Disease: Proof of Concept in an Aggravated Mouse Model.

Fabry disease (FD) is a multisystemic lysosomal storage disorder caused by the loss of α-galactosidase A (α-Gal) function. The current standard of care, enzyme replacement therapies, while effective in reducing kidney pathology when treated early, do not fully ameliorate cardiac issues, neuropathic manifestations, and risk of cerebrovascular events. Adeno-associated virus (AAV)-based gene therapies (AAV-GT) can provide superior efficacy across multiple tissues owing to continuous, endogenous production of the therapeutic enzyme and lower treatment burden.

Effects of reaction environments on the structure and physicochemical properties of chitosan and its derivatives.

The structural transformation of chitosan caused by reaction environment is one of the main factors affecting its functional properties. Herein, the effects of homogeneous and heterogeneous reactions on the structure and properties of chitosan were investigated. The pretreatment of reaction increased the deacetylation degree (DD) of chitosan and resulted in its degradation.

Omics insights into spermatozoa activation induced by Fetal bovine serum in viviparous black rockfish (Sebastes schlegelii).

Black rockfish (Sebastes schlegelii) is an economically important marine species with the characteristics of viviparity. The spermatozoa were transferred into the ovary by mating and stored for several months until fertilization. Little is known about spermatozoa activation and its mechanism in black rockfish.

Homogeneous deacetylation and degradation of chitin in NaOH/urea dissolution system.

Since being discovered, alkali/urea has been widely used in the dissolution of natural polysaccharides and the preparation of functional materials such as hydrogels, fibers, films and nanoparticles. This work will focus on verifying the structural stability, homogeneous degradation and deacetylation of chitin in alkali-soluble systems. The chitin was dissolved in NaOH/urea solution and stored at different temperature.

The transcriptional coactivator Taz regulates proximodistal patterning of the pronephric tubule in zebrafish.

The zebrafish pronephric tubule consists of proximal and distal segments and a collecting duct. The proximal segment is subdivided into the neck, proximal convoluted tubule (PCT) and proximal straight tubule (PST) segments. The distal segment consists of the distal-early (DE) and distal-late (DL) segments.

Early, H+-V-ATPase-dependent proton flux is necessary for consistent left-right patterning of non-mammalian vertebrates.

Biased left-right asymmetry is a fascinating and medically important phenomenon. We provide molecular genetic and physiological characterization of a novel, conserved, early, biophysical event that is crucial for correct asymmetry: H+ flux. A pharmacological screen implicated the H+-pump H+-V-ATPase in Xenopus asymmetry, where it acts upstream of early asymmetric markers.

The small heart mutation reveals novel roles of Na+/K+-ATPase in maintaining ventricular cardiomyocyte morphology and viability in zebrafish.

Forward genetic screens in zebrafish have been used to identify mutations in genes with important roles in organogenesis. One of these mutants, small heart, develops a diminutive and severely malformed heart and multiple developmental defects of the brain, ears, eyes, and kidneys. Using a positional cloning approach, we identify that the mutant gene encodes the zebrafish Na+/K+-ATPase alpha1B1 protein.