Cell and Gene Therapies for Mucopolysaccharidoses: Base Editing and Therapeutic Delivery to the CNS

Although individually uncommon, rare diseases collectively account for a considerable proportion of disease impact worldwide. A group of rare genetic diseases called the mucopolysaccharidoses (MPSs) are characterized by accumulation of partially degraded glycosaminoglycans cellularly. MPS results in...

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Detalles Bibliográficos
Autores principales: Christensen, Chloe L., Ashmead, Rhea E., Choy, Francis Y. M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2019
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6787741/
https://www.ncbi.nlm.nih.gov/pubmed/31248000
http://dx.doi.org/10.3390/diseases7030047

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6787741/
https://www.ncbi.nlm.nih.gov/pubmed/31248000
http://dx.doi.org/10.3390/diseases7030047

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