Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I

Lysosomal storage diseases (LSDs) are rare inherited metabolic disorders characterized by a dysfunction in lysosomes, leading to waste material accumulation and severe organ damage. Enzyme replacement therapy (ERT) and haematopoietic stem cell transplant (HSCT) have been exploited as potential treat...

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Detalles Bibliográficos
Autores principales: Penati, Rachele, Fumagalli, Francesca, Calbi, Valeria, Bernardo, Maria Ester, Aiuti, Alessandro
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Netherlands 2017
Materias:
Ssiem 2016
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5500670/
https://www.ncbi.nlm.nih.gov/pubmed/28560469
http://dx.doi.org/10.1007/s10545-017-0052-4

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5500670/
https://www.ncbi.nlm.nih.gov/pubmed/28560469
http://dx.doi.org/10.1007/s10545-017-0052-4

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