TY - JOUR

T1 - Experimental Models of CNS Disorders Related to Lysosomal Storage Diseases

AU - Калуев, Алан Валерьевич

AU - Лебедев, Андрей Сергеевич

AU - Колесникова, Татьяна Олеговна

AU - Котова, Мария Михайловна

AU - Галстян, Давид Самвелович

N1 - Lebedev, A.S., Kotova, M.M., Kolesnikova, T.O. et al. Experimental Models of CNS Disorders Related to Lysosomal Storage Diseases. J Evol Biochem Phys 59, 2072–2085 (2023). https://doi.org/10.1134/S0022093023060145

PY - 2024/1/9

Y1 - 2024/1/9

N2 - Lysosomal storage diseases (LSDs) are caused by enzyme deficiency in the cellular lysosomal apparatus, leading to a pathological accumulation of undigested cellular material (proteins, lipids or carbohydrates) and eventual tissue damage. Clinically and etiologically diverse, this group includes over 70 presently recognized hereditary conditions that have no effective therapy known to date. Most common manifestations of LSDs are brain lesions leading to various neurological deficits. Thus, the search for therapeutic strategies targeting these disorders represents an urgent unmet biomedical task, also necessitating the use of appropriate and valid experimental (animal) models. Here, we discuss the available models of LSDs and the applicability of rodents and zebrafish as model organisms for probing these pathologies.

AB - Lysosomal storage diseases (LSDs) are caused by enzyme deficiency in the cellular lysosomal apparatus, leading to a pathological accumulation of undigested cellular material (proteins, lipids or carbohydrates) and eventual tissue damage. Clinically and etiologically diverse, this group includes over 70 presently recognized hereditary conditions that have no effective therapy known to date. Most common manifestations of LSDs are brain lesions leading to various neurological deficits. Thus, the search for therapeutic strategies targeting these disorders represents an urgent unmet biomedical task, also necessitating the use of appropriate and valid experimental (animal) models. Here, we discuss the available models of LSDs and the applicability of rodents and zebrafish as model organisms for probing these pathologies.

UR - https://www.mendeley.com/catalogue/883ad69c-17b3-3d81-a698-31f4a3efdb34/

U2 - 10.1134/s0022093023060145

DO - 10.1134/s0022093023060145

M3 - Article

VL - 59

SP - 2072

EP - 2085

JO - Journal of Evolutionary Biochemistry and Physiology

JF - Journal of Evolutionary Biochemistry and Physiology

SN - 0022-0930

IS - 6

ER -