Filter the results Item type Select All/None News Item Researcher Page Publication File Event Image Folder Collection New items since Yesterday Last week Last month Ever Search results Subscribe to an always-updated RSS feed. 26 items matching your search terms. Sort by relevance date (newest first) alphabetically Esrrb guides naive pluripotent cells through the formative transcriptional programme Nature Cell Biology Located in Newsroom / Publications Genome editing without nucleases confers proliferative advantage to edited hepatocytes and corrects Wilson disease JCI Insight Located in Newsroom / Publications Increased expression or activation of TRPML1 reduces hepatic storage of toxic Z alpha-1 antitrypsin Molecular Therapy Located in Newsroom / Publications Integrated exome and transcriptome analysis prioritizes MAP4K4 de novo frameshift variants in autism spectrum disorder as a novel disease–gene association Human Genetics Located in Newsroom / Publications Investigation on lysosomal accumulation by a quantitative analysis of 2D phase-maps in digital holography microscopy Journial of Quantitative Cell Science - Citometry Located in Newsroom / Publications Liver-directed gene therapy for ornithine aminotransferase deficiency EMBO Mol Med Located in Newsroom / Publications Loss of the batten disease protein CLN3 leadsto mis-trafficking of M6PR and defective autophagic-lysosomal reformation Nature Communications Located in Newsroom / Publications Lysosomes as coordinators of cellular catabolism, metabolic signalling and organ physiology Nature reviews Located in Newsroom / Publications New mouse models with hypomorphic SUMF1 variants mimic attenuated forms of multiple sulfatase deficiency Journal of Inherited Metabolic Disease Located in Newsroom / Publications Pharmacological potentiators of the calcium signaling cascade identified by high-throughput screening PNAS Nexus Located in Newsroom / Publications Previous 10 items 1 2 3 Next 6 items