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Chemical and Molecular Strategies in Restoring Autophagic Flux in TDP-43 Proteinopathy. [PDF]

Molecules
Jamerlan A, Hulme J.
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Rethinking lysosomes and lysosomal disease

Neuroscience Letters, 2021
Lysosomal storage diseases were recognized and defined over a century ago as a class of disorders affecting mostly children and causing systemic disease often accompanied by major neurological consequences. Since their discovery, research focused on understanding their causes has been an important driver of our ever-expanding knowledge of cell biology ...
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LYSOSOMAL STORAGE DISEASES

Neuropathology and Applied Neurobiology, 1978
The majority of lysosomal storage diseases affect the central nervous system. Those that reflect a primary lysosomal disorder are associated with genetically determined deficiencies of specific lysosomal enzymes and storage of the relevant substrate. Autofluorescent lipopigments accumulate in the ceroid‐lipofuscinoses, a heterogeneous group of diseases
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Lysosomal storage diseases

Current Treatment Options in Neurology, 2001
Lysosomal storage disorders (LSDs), over 40 different diseases, are now considered treatable disorders. Only a few short years ago, Lysosomal storage disorders were seen as interesting neurodegenerative disorders without any potential for treatment. Effective treatment strategies such as bone marrow transplantation (BMT), enzyme replacement therapy ...
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