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| http://dx.doi.org/10.1136/oem.3.3.183 | DOI Listing |
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Preclinical use of a clinically-relevant scAAV9/SUMF1 vector for the treatment of multiple sulfatase deficiency.
Commun Med (Lond)
January 2025
Rare Disease Translational Center, The Jackson Laboratory, Bar Harbor, ME, USA.
Background: Multiple Sulfatase Deficiency (MSD) is a rare inherited lysosomal storage disorder characterized by loss of function mutations in the SUMF1 gene that manifests as a severe pediatric neurological disease. There are no available targeted therapies for MSD.
Methods: We engineered a viral vector (AAV9/SUMF1) to deliver working copies of the SUMF1 gene and tested the vector in Sumf1 knock out mice that generally display a median lifespan of 10 days.
InterDIA: Interpretable Prediction of Drug-induced Autoimmunity through Ensemble Machine Learning Approaches.
Toxicology
January 2025
Deparment of clinical pharmacy, Jieyang People's Hospital, 522000, China. Electronic address:
Drug-induced autoimmunity (DIA) is a non-IgE immune-related adverse drug reaction that poses substantial challenges in predictive toxicology due to its idiosyncratic nature, complex pathogenesis, and diverse clinical manifestations. To address these challenges, we developed InterDIA, an interpretable machine learning framework for predicting DIA toxicity based on molecular physicochemical properties. Multi-strategy feature selection and advanced ensemble resampling approaches were integrated to enhance prediction accuracy and overcome data imbalance.
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Plant Cell Rep
January 2025
Department of Biophysics, Molecular Biology and Bioinformatics, University of Calcutta, 92, APC Road, Kolkata, 700 009, India.
Melatonin increases Pb tolerance in P. ovata seedlings via the regulation of growth and stress-related phytohormones, ROS scavenging and genes responsible for melatonin synthesis, metal chelation, and stress defense. Lead (Pb) is a highly toxic heavy metal that accumulates in plants through soil and air contamination and impairs its plant growth and development.
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View Article and Find Full Text PDFFluoride Exposure Modulates Skeletal Development and Mineralization in Zebrafish Larvae.
Environ Toxicol
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Molecular Toxicology Laboratory, Department of Biotechnology, Bharathiar University, Coimbatore, Tamil Nadu, India.
The presence of high levels of fluoride (F) in groundwater is a major issue worldwide. Although F is essential for healthy teeth and bones, excessive exposure can cause fluorosis or F toxicity. This condition primarily affects the hard tissues due to their high F retention capacity.
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