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lüll Gene Therapy Approaches to Treat the Neurodegeneration and Visual Failure in Neuronal Ceroid Lipofuscinoses Kleine Holthaus SM; Smith AJ; Mole SE; Ali RRAdv Exp Med Biol 2018[]; 1074 (ä): 91-99Neuronal ceroid lipofuscinoses (NCLs) are a group of fatal, inherited lysosomal storage disorders mostly affecting the central nervous system of children. Symptoms include vision loss, seizures, motor deterioration and cognitive decline ultimately resulting in premature death. Studies in animal models showed that the diseases are amenable to gene supplementation therapies, and over the last decade, major advances have been made in the (pre)clinical development of these therapies. This mini-review summarises and discusses current gene therapy approaches for NCL targeting the brain and the eye.|Animals[MESH]|Brain/enzymology[MESH]|Child[MESH]|Clinical Trials as Topic[MESH]|Dependovirus/genetics[MESH]|Disease Models, Animal[MESH]|Genetic Therapy/*methods[MESH]|Genetic Vectors/administration & dosage/therapeutic use[MESH]|Humans[MESH]|Infant[MESH]|Injections, Intraocular[MESH]|Injections, Intraventricular[MESH]|Lysosomes/enzymology[MESH]|Nerve Degeneration/*therapy[MESH]|Neuronal Ceroid-Lipofuscinoses/complications/enzymology/*therapy[MESH]|Organ Specificity[MESH]|Vision Disorders/etiology/*therapy[MESH] |