Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA

Mucopolysaccharidosis type IIIA (MPS-IIIA) is a lysosomal storage disorder (LSD) caused by inherited defect of sulfamidase, a lysosomal sulfatase. MPS-IIIA is one of the most common and severe forms of LSDs with CNS involvement. Presently there is no cure. Here we have developed a new gene delivery...

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Veröffentlicht in:Mol Ther Methods Clin Dev
Hauptverfasser: Sorrentino, Nicolina Cristina, Cacace, Vincenzo, De Risi, Maria, Maffia, Veronica, Strollo, Sandra, Tedesco, Novella, Nusco, Edoardo, Romagnoli, Noemi, Ventrella, Domenico, Huang, Yan, Liu, Nan, Kalled, Susan L., Choi, Vivian W., De Leonibus, Elvira, Fraldi, Alessandro
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society of Gene & Cell Therapy 2019
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6881609/
https://ncbi.nlm.nih.gov/pubmed/31788497
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.10.009
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