Factors influencing transfection efficiency of pIDUA/nanoemulsion complexes in a mucopolysaccharidosis type I murine model
نویسندگان
چکیده
منابع مشابه
Factors influencing transfection efficiency of pIDUA/nanoemulsion complexes in a mucopolysaccharidosis type I murine model
Mucopolysaccharidosis type I (MPS I) is an autosomal disease caused by alpha-l-iduronidase (IDUA) deficiency. This study used IDUA knockout mice as a model to evaluate whether parameters such as dose of plasmid and time of treatment could influence the transfection efficiency of complexes formed with PEGylated cationic nanoemulsions and plasmid (pIDUA), which contains the gene that encodes for ...
متن کاملEffects of Enzyme Replacement Therapy Started Late in a Murine Model of Mucopolysaccharidosis Type I
Mucopolysaccharidosis type I (MPS I) is a progressive disorder caused by deficiency of α-L-iduronidase (IDUA), which leads to storage of heparan and dermatan sulphate. It is suggested that early enzyme replacement therapy (ERT) leads to better outcomes, although many patients are diagnosed late and don't receive immediate treatment. This study aims to evaluate the effects of late onset ERT in a...
متن کاملMurine Mucopolysaccharidosis Type
We have characterized a new mutant mouse that has virtually no /-glucuronidase activity. This biochemical defect causes a murine lysosomal storage disease that has many interesting similarities to human mucopolysaccharidosis type VII (MPS VII; Sly syndrome; fl-glucuronidase deficiency). Genetic analysis showed that the mutation is inherited as an autosomal recessive that maps to the fl-glucuron...
متن کاملan investigation on influencing factors on tourists shopping attitude of iranian handmade carpet in isfahan
چکیده ندارد.
15 صفحه اولCharacterization of a murine model of mucopolysaccharidosis type IIID: A knockout model
The Mucopolysaccharidoses (MPS) are a class of lysosomal storage disorders, characterized by the primary lysosomal storage of either single or multiple species of glycosaminoglycan (GAG) which leads to cell, tissue, and organ dysfunction. This is manifested clinically by either bone/connective tissue disease, central nervous system (CNS) disease, or both, and often results in premature death. M...
متن کاملذخیره در منابع من
با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید
ژورنال
عنوان ژورنال: International Journal of Nanomedicine
سال: 2017
ISSN: 1178-2013
DOI: 10.2147/ijn.s121558