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Gene therapy for lysosomal storage diseases: Current clinical trial prospects

Lysosomal storage diseases (LSDs) are a group of metabolic inborn errors caused by defective enzymes in the lysosome, resulting in the accumulation of undegraded substrates. LSDs are progressive diseases that exhibit variable rates of progression depending on the disease and the patient. The availab...

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Detalles Bibliográficos
Autores principales:	Kido, Jun, Sugawara, Keishin, Nakamura, Kimitoshi
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2023
Materias:	Genetics
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9880060/ https://www.ncbi.nlm.nih.gov/pubmed/36713078 http://dx.doi.org/10.3389/fgene.2023.1064924

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