Long-term reestablishment of alpha-L-iduronidase activity in MPS I fibroblasts after non-viral gene transfer

Long-term reestablishment of alpha-L-iduronidase activity in MPS I fibroblasts after non-viral gene transfer

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Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disorder due to deficiency of alpha-L-iduronidase (IDUA). Limitations such as need of weekly injection, high morbidity and mortality  and high cost of the current treatments show the need for new approaches to treat this disease. In this wo...

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Bibliographic Details
Main Authors: Fabiano de Oliveira Poswar, Fabiana Quoos Mayer, Maira Graeff Burin, Ursula da Silveira Matte, Roberto Giugliani, Guilherme Baldo
Format: Article
Language:English
Published: Hospital de Clinicas de Porto Alegre ; Universidade Federal do Rio Grande do Sul (UFRGS) 2017-12-01
Series:Clinical and Biomedical Research
Subjects:
Mucopolysaccharidosis I
genetic therapy
iduronidase.
Online Access:http://seer.ufrgs.br/hcpa/article/view/74048

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