The Impact of QSP Modeling on the Design and Optimization of Gene Therapy Approaches. [PDF]
ABSTRACT Quantitative Systems Pharmacology (QSP) is increasingly utilized to support the design and translation of gene therapies. This perspective outlines the application of QSP modeling across three domains of gene therapy: mRNA‐based therapeutics, adeno‐associated virus (AAV) vectors, and genome editing systems.
Rayad N, Chowdhury EA, Meno-Tetang GML.
europepmc +2 more sources
Phospholipase C Produced by Clostridium botulinum Types C and D:Comparison of Gene, Enzymatic, and Biological Activities with Those of Clostridium perfringens Alpha-toxin [PDF]
Clostridium botulinum type C and D strains recently have been found to produce PLC on egg yolk agar plates. To characterize the gene, enzymatic and biological activities of C.
Fatmawati, Ni Nengah Dwi +8 more
core +1 more source
Acid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disorder with heterogeneous clinical manifestations, including hepatosplenomegaly and infiltrative pulmonary disease, and is associated with significant morbidity and mortality ...
Chanchala D. Kaddi +15 more
doaj +1 more source
On the role of bioactive sphingolipids and their metabolizing enzymes in cancer [PDF]
Ceramide is a family of closely related molecules, which are presumed to be in the center of sphingolipid metabolism. Among sphingolipid metabolites, several ceramide subspecies and sphingosine induce apoptosis, cell cycle arrest and death, whereas ...
Ahmed, Dilruba
core +1 more source
Sphingolipids and cholesterol modulate membrane susceptibility to cytosolic phospholipase A2
Modulation of cytosolic phospholipase A2 (cPLA2) activity by sphingomyelin (SPH), ceramide (Cer), and cholesterol (Chol) was investigated in CHO-2B cells activated by the calcium ionophore A23187 and epinephrine.
Elsa Klapisz +4 more
doaj +1 more source
Mechanisms of Action and Cell Death Associated with Clostridium perfringens Toxins. [PDF]
Clostridium perfringens uses its large arsenal of protein toxins to produce histotoxic, neurologic and intestinal infections in humans and animals. The major toxins involved in diseases are alpha (CPA), beta (CPB), epsilon (ETX), iota (ITX), enterotoxin (
McClane, Bruce A +2 more
core +3 more sources
Effects of flavonoids on sphingolipid turnover in the toxin-damaged liver and liver cells [PDF]
Background The ceramide generation is an early event in the apoptotic response to numerous stimuli including the oxidative stress and ceramide analogs mimic the stress effect and induce apoptosis.
Nataliya A Babenko, Elena G Shakhova
core +2 more sources
The Extracellular Vesicles of the Helminth Pathogen, Fasciola hepatica: Biogenesis Pathways and Cargo Molecules Involved in Parasite Pathogenesis [PDF]
Extracellular vesicles (EVs) released by parasites have important roles in establishing and maintaining infection. Analysis of the soluble and vesicular secretions of adult Fasciola hepatica has established a definitive characterization of the total ...
Bernal, Dolores +11 more
core +1 more source
Les sphingolipides : vecteurs d’agents pathogènes et cause de maladies génétiques [PDF]
Les sphingolipides (SPL) sont des molécules ubiquitaires indispensables au maintien et au développement des organismes vivants. Ils ne sont pas répartis uniformément le long de la membrane mais regroupés sous forme de microdomaines lipidiques appelés ...
Fasano, Caroline +3 more
core +1 more source
Abstract Background and Aims High cholesterol absorption efficiency is determined by genetic variation in small intestinal sterol transporters and affects one‐third of individuals. Their risk for atherosclerotic cardiovascular disease (ASCVD) is increased compared with low cholesterol absorbers, despite similar serum lipid concentrations.
Katariina Öörni +7 more
wiley +1 more source

