Results 121 to 130 of about 249,219 (350)
Drug Design, Development and Therapy, 2017 David AH Whiteman,* Alan Kimura* Research & Development, Shire Human Genetic Therapies, Inc., Lexington, MA, USA *These authors contributed equally to this work Abstract: Mucopolysaccharidosis type II (MPS II; Hunter syndrome; OMIM 309900) is
Whiteman DAH, Kimura A
doaj
In Situ Activation of Human Erythrocyte Prolidase: Potential for Enzyme Replacement Therapy in Prolidase Deficiency [PDF]
, 1988 Peter Hechtman +3 more
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LIN28B Promotes Cancer Cell Dissemination and Angiogenesis
Advanced Biology, EarlyView.Children diagnosed with high‐risk neuroblastoma have a 5‐year event‐free survival rate of less than 50% and poor outcomes after recurrence. Deregulation of the LIN28B oncogene can be addressed in these patients. Upregulation of LIN28B is shown to support the metastatic cascade.
Diana Corallo +8 more
wiley +1 more source
Enzyme replacement therapy in porphyrias—IV. First successful human clinical trial of δ-aminolevulinate dehydratase-loaded erythrocyte ghosts [PDF]
, 1983 Alcira Batlle +5 more
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A nonhuman primate model of inherited retinal disease. [PDF]
, 2019 Inherited retinal degenerations are a common cause of untreatable blindness worldwide, with retinitis pigmentosa and cone dystrophy affecting approximately 1 in 3500 and 1 in 10,000 individuals, respectively.
Artemyev, Nikolai O +21 more
core
Advanced Biology, EarlyView.In vitro cancer models are advantageous for studying important processes such as tumorigenesis, cancer growth, invasion, and metastasis. The complexity and biological relevance increase depending on the model structure, organization, and composition of materials and cells.
Kyndra S. Higgins +2 more
wiley +1 more source
Letter to the Editor: Effectiveness of HLA-Compatible Fibroblasts for Enzyme Replacement Therapy in the Mucopolysaccharidoses [PDF]
, 1982 Mark Dean +3 more
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Early initiation of enzyme replacement therapy for the mucopolysaccharidoses
Molecular Genetics and Metabolism, 2014 The mucopolysaccharidoses (MPS), a group of rare genetic disorders caused by defects in glycosaminoglycan (GAG) catabolism, are progressive, multi-systemic diseases with a high burden of morbidity. Enzyme replacement therapy (ERT) is available for MPS I, II, and VI, and may improve walking ability, endurance, and pulmonary function as evidenced by data
openaire +2 more sources
Advanced Biology, EarlyView.The novelty of this study showed that the injection of exosomes produced from ADMSCs in combination with Roflumilast poses a more favorable therapeutic outcome for CKD induced by Adriamycin, compared to therapy with exosomes or Roflumilast alone. Roflumilast and exosomes treatment lowered the expression of the apoptotic, fibrotic, and inflammatory ...
Mohamed Ali +5 more
wiley +1 more source
Enzyme Replacement Therapy Improves Reproductive Performance in Mucopolysaccharidosis Type VII Mice But Does Not Prevent Postnatal Losses [PDF]
, 1999 Brian W. Soper +5 more
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