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CRISPR/Cas-Based Ex Vivo Gene Therapy and Lysosomal Storage Disorders: A Perspective Beyond Cas9

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Myosin VI controls localization of Golgi satellites at active presynaptic boutonsPreprint

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Plasma Membrane Remodelling in GM2 Gangliosidoses Drives Synaptic DysfunctionPreprint

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Cathepsin B inhibition blocks amyloidogenesis in the mouse models of neurological lysosomal diseases MPS IIIC and sialidosis

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Reactivation of mTOR signaling slows neurodegeneration in a lysosomal sphingolipid storage disease

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Cerebellar and Midbrain Lysosomal Enzyme Deficiency in Isolated Dystonia

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Glycoengineering for the Production of Lysosomal Enzymes

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Glycoengineering for the Production of Lysosomal Enzymes

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Cardiac affection in lysosomal storage disorders and effect of enzyme replacement therapy

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Suspected vinca alkaloid toxicosis in a 9‐week‐old dachshund puppy due to ingestion of periwinkle bush ( Catharanthus spp., formerly Vinca spp.)

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Synthetic analogue of adrenocorticotropic hormone, ACTH (4-7) PGP delays neurological manifestations in diseases of mucopolysaccharidosis III spectrum by reducing neuroinflammation and rescuing neurotransmission, synaptogenesis, and axonal demyelinationPreprint

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Naturally occurring lysosomal storage disease consistent with neuronal ceroid lipofuscinosis in a group of 5 related captive rhesus macaques ( Macaca mulatta )

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Biologic Therapies for Alleviating Neurodegeneration in Lysosomal Storage Diseases

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Synaptic Function and Dysfunction in Lysosomal Storage Diseases

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