Henke, Larissa; Ghorbani, Ali; Mole, Sara E; (2025) The use of nanocarriers in treating Batten disease: A systematic review. International Journal of Pharmaceutics , 670 , Article 125094. 10.1016/j.ijpharm.2024.125094.

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Abstract

The neuronal ceroid lipofuscinoses, commonly known as Batten disease, are a group of lysosomal storage disorders affecting children. There is extensive central nervous system and retinal degeneration, resulting in seizures, vision loss and a progressive cognitive and motor decline. Enzyme replacement and gene therapies are being developed, and mRNA and oligonucleotide therapies are more recently being considered. Overcoming the challenges of the blood–brain barrier and blood-ocular barrier is crucial for effectively targeting the brain and eye, whatever the therapeutic approach. Nanoparticles and extracellular vesicles are small carriers that can encapsulate a cargo and pass through these cell barriers. They have been investigated as drug carriers for other pathologies and could be a promising treatment strategy for Batten disease. Their use in gene, enzyme, or mRNA replacement therapy of all lysosomal storage disorders, including Mucopolysaccharidoses, Niemann-Pick diseases, and Fabry disease, is investigated in this systematic review. Different nanocarriers can efficiently target the lysosome and cross the barriers into the brain and eyes. This supports continued exploration of nanocarriers as potential future treatment options for Batten disease.

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