A new strategy aims to improve gene therapy for Pompe disease by optimizing both the genetic component that restores the function of a deficient lysosomal enzyme and the vector that delivers it to the target tissue while avoiding the liver. The findings suggest that combining an optimized transgene with a targeted capsid could significantly enhance the effectiveness of gene therapy for Pompe disease.
University of Florida researchers have synthesized proteolysis targeting chimeras (PROTACs) comprising an E3 ubiquitin ligase-binding moiety coupled to Bcl-2-like protein 1 (Bcl-xl; Bcl-X; BCL2L1)- and apoptosis regulator Bcl-2-targeting moiety.
Hypoxia-inducible factors (HIFs) play a central role in how retinal cells respond to low oxygen, but their chronic activation has been linked to the development of age-related macular degeneration (AMD) and related retinal disorders.
Mayo Foundation for Medical Education and Research (MFMER) and University of Florida have identified atrial natriuretic peptide B (NPR2; guanylate cyclase B) receptor positive allosteric modulators (PAMs) reported to be useful for the treatment of fibrosis.
Work at the University of Florida has led to the identification of nuclear receptor subfamily 1 group D member 1 (Rev-erbA-α) antagonists reported to be useful for the treatment of cancer, sarcopenia and Duchenne muscular dystrophy.
Researchers at the University North Carolina at Chapel Hill and University of Florida have divulged triazoles acting as κ-opioid receptor agonists reported to be useful for the treatment of pain, pruritus, depression, inflammation and more.
The University of Florida has prepared and tested PPARγ agonists reported to be useful for the treatment of diabetes, hyperparathyroidism, metabolic syndrome, multiple myeloma, nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH), obesity, osteoporosis and Paget disease, among others.
University of Florida has identified σ receptor ligands reported to be useful for the treatment of cancer, depression, neurodegeneration, stroke, pain, multiple sclerosis, schizophrenia and anxiety.
Researchers from the University of Florida have developed a novel mRNA cancer vaccine that aims to reprogram the immune system to target and attack cancer cells effectively. The study led by Elias Sayour reported a new mRNA vaccine design where the mRNA payload was wrapped into a multilamellar vesicle within an onion-like hierarchical structure. The findings were published in the May 9, 2024 issue of Cell.
The University of Florida Health Proton Therapy Institute’s director of Physics, Perry Johnson, has filed for protection of a method for patient posture and alignment using mixed reality (MixR) visualization.
