Elements of lentiviral vector design toward gene therapy for treating mucopolysaccharidosis I

Mucopolysaccharidosis type I (MPS I) is a lysosomal disease caused by α-l-iduronidase (IDUA) deficiency and accumulation of glycosaminoglycans (GAG). Lentiviral vector encoding correct IDUA cDNA could be used for treating MPS I. To optimize the lentiviral vector design, 9 constructs were designed by...

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Detalles Bibliográficos
Autores principales: Ou, Li, Przybilla, Michael J., Koniar, Brenda L., Whitley, Chester B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2016
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4987501/
https://www.ncbi.nlm.nih.gov/pubmed/27556013
http://dx.doi.org/10.1016/j.ymgmr.2015.11.004

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