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Lentivirus-mediated gene therapy for Fabry disease: 5-year End-of-Study results from the Canadian FACTs trial.
Clin Transl Med
January 2025
Department of Pediatrics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
Background: Fabry disease is an X-linked lysosomal storage disorder due to a deficiency of α-galactosidase A (α-gal A) activity. Our goal was to correct the enzyme deficiency in Fabry patients by transferring the cDNA for α-gal A into their CD34+ hematopoietic stem/progenitor cells (HSPCs). Overexpression of α-gal A leads to secretion of the hydrolase; which can be taken up and used by uncorrected bystander cells.
View Article and Find Full Text PDFFacts and hopes in neoadjuvant immunotherapy combinations in resectable non-small-cell lung cancer.
Clin Cancer Res
January 2025
University Hospital Essen, Essen, Germany.
Antibodies targeting immune checkpoints, such as PD-1, PD-L1, or CTLA-4, have transformed the treatment of patients with lung cancers. Unprecedented rates of durable responses are achieved in an imperfectly characterized population of patients with metastatic disease. More recently, immune checkpoint inhibitors have been explored in patients with resectable non-small-cell lung cancers.
View Article and Find Full Text PDFLacustrine sedimentation patterns at the Northern Antarctic Peninsula and surroundings as a response to late Holocene and Modern Climate changes.
Sci Rep
December 2024
Soil and Water Management & Crop Nutrition Laboratory, Joint FAO/IAEA Centre of Nuclear Techniques in Food and Agriculture, International Atomic Energy Agency, Vienna, Austria.
The Northern Antarctic Peninsula (NAP) and the West Antarctic Ice Sheet (WAIS) are likely to respond rapidly to climate changes by increasing the collapse of peripheral ice shelves and the number of days above 0 °C. These facts make this region a representative hotspot of the global sea level rise and the location of one of the global climate tipping points (thresholds in the Earth system whose changes may become irreversible, if exceeded). Understanding the climate evolution of the NAP, based on past evidences, may help infer its future scenario.
View Article and Find Full Text PDFStability-Indicating Liquid Chromatographic Method Development for the Simultaneous Determination of Amitriptyline Hydrochloride and Propranolol Hydrochloride in Tablet Dosage Form.
J Chromatogr Sci
January 2025
Department of Pharmaceutical Chemistry, JSS College of Pharmacy, JSS Academy of Higher Education & Research, Mysuru 570 015, Karnataka, India.
The combination of the tricyclic antidepressant amitriptyline hydrochloride (AMH) and the non-selective beta-adrenergic blocker propranolol hydrochloride (PPH) is used for migraine prophylaxis. Higher doses of AMH trigger cardiac arrhythmias, anxiety, tachycardia, convulsions, hyperglycemia and anticholinergic side effects. The combined dosage formulation of AMH and PPH leads to drug-drug interactions; causes sedation, xerostomia, dysuria, insomnia and bradycardia; and results in patient non-compliance.
View Article and Find Full Text PDFHow proteins manage to fold and how chaperones manage to assist the folding.
Phys Life Rev
December 2024
Institute of Protein Research, Russian Academy of Sciences, Pushchino, Moscow Region, 142290, Russian Federation. Electronic address:
This review presents the current understanding of (i) spontaneous self-organization of spatial structures of protein molecules, and (ii) possible ways of chaperones' assistance to this process. Specifically, we overview the most important features of spontaneous folding of proteins (mostly, of the single-domain water-soluble globular proteins): the choice of the unique protein structure among zillions of alternatives, the nucleation of the folding process, and phase transitions within protein molecules. We consider the main experimental facts on protein folding, both in vivo and in vitro, of both kinetic and thermodynamic nature.
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