Long-term reestablishment of alpha-L-iduronidase activity in MPS I fibroblasts after non-viral gene transfer
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...
Main Authors: | , , , , , |
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Format: | Article |
Language: | English |
Published: |
Hospital de Clinicas de Porto Alegre ; Universidade Federal do Rio Grande do Sul (UFRGS)
2017-12-01
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Series: | Clinical and Biomedical Research |
Subjects: | |
Online Access: | http://seer.ufrgs.br/index.php/hcpa/article/view/74048 |