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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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Bibliografski detalji
Izdano u:Diseases
Glavni autori: Christensen, Chloe L., Ashmead, Rhea E., Choy, Francis Y. M.
Format: Artigo
Jezik:Inglês
Izdano: MDPI 2019
Teme:
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6787741/
https://ncbi.nlm.nih.gov/pubmed/31248000
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/diseases7030047
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