Extracellular Vesicles as Drug Carriers for Enzyme Replacement Therapy to Treat CLN2 Batten Disease: Optimization of Drug Administration Routes

CLN2 Batten disease (BD) is one of a broad class of lysosomal storage disorders that is characterized by the deficiency of lysosomal enzyme, TPP1, resulting in a build-up of toxic intracellular storage material in all organs and subsequent damage. A major challenge for BD therapeutics is delivery of...

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Detalles Bibliográficos
Autores principales: Haney, Matthew J., Zhao, Yuling, Jin, Yeon S., Batrakova, Elena V.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7290714/
https://www.ncbi.nlm.nih.gov/pubmed/32443895
http://dx.doi.org/10.3390/cells9051273

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7290714/
https://www.ncbi.nlm.nih.gov/pubmed/32443895
http://dx.doi.org/10.3390/cells9051273

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