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Current and Emerging Therapies for Lysosomal Storage Disorders.
Drugs
January 2025
Lysosomal Storage Disorders Unit, Royal Free London NHS Foundation Trust, University College London, London, NW3 2QG, UK.
Lysosomal storage disorders (LSDs) are rare inherited metabolic disorders characterized by defects in the function of specific enzymes responsible for breaking down substrates within cellular organelles (lysosomes) essential for the processing of macromolecules. Undigested substrate accumulates within lysosomes, leading to cellular dysfunction, tissue damage, and clinical manifestations. Clinical features vary depending on the degree and type of enzyme deficiency, the type and extent of substrate accumulated, and the tissues affected.
View Article and Find Full Text PDFState-of-the-Art Liver Cancer Organoids: Modeling Cancer Stem Cell Heterogeneity for Personalized Treatment.
BioDrugs
January 2025
Orsay-Vallée Campus, Paris-Saclay University, Gif-sur-Yvette, France.
Liver cancer poses a global health challenge with limited therapeutic options. Notably, the limited success of current therapies in patients with primary liver cancers (PLCs) may be attributed to the high heterogeneity of both hepatocellular carcinoma (HCCs) and intrahepatic cholangiocarcinoma (iCCAs). This heterogeneity evolves over time as tumor-initiating stem cells, or cancer stem cells (CSCs), undergo (epi)genetic alterations or encounter microenvironmental changes within the tumor microenvironment.
View Article and Find Full Text PDFNatural products and long non-coding RNAs in prostate cancer: insights into etiology and treatment resistance.
Naunyn Schmiedebergs Arch Pharmacol
January 2025
Department of Biochemistry, Faculty of Pharmacy, Badr University in Cairo (BUC), Badr City, , 11829, Cairo, Egypt.
Globally, the incidence and death rates associated with cancer persist in rising, despite considerable advancements in cancer therapy. Although some malignancies are manageable by a mix of chemotherapy, surgery, radiation, and targeted therapy, most malignant tumors either exhibit poor responsiveness to early identification or endure post-treatment survival. The prognosis for prostate cancer (PCa) is unfavorable since it is a perilous and lethal malignancy.
View Article and Find Full Text PDFPilomatrix carcinoma unveiled: a systematic review of clinical characteristics, pathogenesis, diagnostic puzzles, and management strategies.
Arch Dermatol Res
January 2025
Premier Dermatology, Ashburn, VA, USA.
Pilomatrix carcinoma (PC) is a rare malignant adnexal tumor originating from follicular matrix cells primarily impacting Caucasian males. This review provides a comprehensive analysis of scientific literature on PC through an exploration of 206 cases reported between 1980 and 2024. We discuss the epidemiology, clinical presentation, histopathology, and diagnostic challenges of PC, and explore various treatment methods for this rare malignancy as well as their associated outcomes.
View Article and Find Full Text PDFKineococcus halophytocola sp. nov., Isolated from Leaves of Halophyte Sesuvium portulacastrum L.
Curr Microbiol
January 2025
Department of Microbiology, Faculty of Science, Kasetsart University, Chatuchak, Bangkok, 10900, Thailand.
An aerobic, Gram-stain-positive, motile, coccus-shaped actinomycete, designated strain LSe6-4, was isolated from leaves of sea purslane (Sesuvium portulacastrum L.) in Thailand and subjected to a polyphasic taxonomic studies. Growth of the strain occurred at temperatures between 15 and 38 °C, and with NaCl concentrations 0-13%.
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