Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA-Reference-Cited by-同舟云学术

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

Published:2019-12 Issue: Volume:15 Page:333-342
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

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

Funder

Shire

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference28 articles.

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4. Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches;Fraldi;Ital. J. Pediatr.,2018

5. Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges;Mingozzi;Nat. Rev. Genet.,2011

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