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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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Principais autores: 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
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2019
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Article
Acesso em linha: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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