Found: 16
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Enzyme Replacement Therapy for Cognitive Decline in Mucopolysaccharidosis Type I: Past, Present, and Future.
- Published in:
- Current Medical Literature: Lysosomal Storage Disease, 2011, v. 9, n. 1, p. 9
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- Publication type:
- Article
Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy.
- Published in:
- 2018
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- Publication type:
- journal article
Intra-Articular AAV9 α-l-Iduronidase Gene Replacement in the Canine Model of Mucopolysaccharidosis Type I.
- Published in:
- Advances in Cell & Gene Therapy, 2023, p. 1, doi. 10.1155/2023/7419017
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- Publication type:
- Article
Evaluation of artificial signal peptides for secretion of two lysosomal enzymes in CHO cells.
- Published in:
- Biochemical Journal, 2021, v. 478, n. 12, p. 2309, doi. 10.1042/BCJ20210015
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- Publication type:
- Article
Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of a-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts.
- Published in:
- Biochemical Journal, 2014, v. 458, n. 2, p. 281, doi. 10.1042/BJ20130845
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- Publication type:
- Article
Quantitative diffusion tensor imaging analysis does not distinguish pediatric canines with mucopolysaccharidosis I from control canines.
- Published in:
- Neuroradiology Journal, 2017, v. 30, n. 5, p. 454, doi. 10.1177/1971400917718844
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- Publication type:
- Article
Diffusion tensor imaging tensor shape analysis for assessment of regional white matter differences.
- Published in:
- Neuroradiology Journal, 2017, v. 30, n. 4, p. 324, doi. 10.1177/1971400917709628
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- Publication type:
- Article
Myelin and Lipid Composition of the Corpus Callosum in Mucopolysaccharidosis Type I Mice.
- Published in:
- Lipids, 2020, v. 55, n. 6, p. 627, doi. 10.1002/lipd.12261
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- Publication type:
- Article
Elevated cerebral spinal fluid biomarkers in children with mucopolysaccharidosis I-H.
- Published in:
- Scientific Reports, 2016, p. 38305, doi. 10.1038/srep38305
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- Publication type:
- Article
Mucopolysaccharidoses type I gene therapy.
- Published in:
- Journal of Inherited Metabolic Disease, 2021, v. 44, n. 5, p. 1088, doi. 10.1002/jimd.12414
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- Publication type:
- Article
Postmortem diagnosis of very long chain acyl‐CoA dehydrogenase (VLCAD) deficiency in a neonate with sudden cardiac death.
- Published in:
- 2023
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- Publication type:
- Case Study
Fatal COVID‐19 infection in a patient with long‐chain 3‐hydroxyacyl‐CoA dehydrogenase deficiency: A case report.
- Published in:
- Journal of Inherited Metabolic Disease Reports, 2020, v. 56, n. 1, p. 40, doi. 10.1002/jmd2.12165
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- Publication type:
- Article
Community Partnership in Precision Medicine: Themes from a Community Engagement Conference.
- Published in:
- 2018
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- Publication type:
- journal article
Biochemical characterization of fluorescent-labeled recombinant human alpha- l-iduronidase in vitro.
- Published in:
- Biotechnology & Applied Biochemistry, 2011, v. 58, n. 6, p. 391, doi. 10.1002/bab.52
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- Publication type:
- Article
Phenotypic expansion of KMT2D‐related disorder: Beyond Kabuki syndrome.
- Published in:
- American Journal of Medical Genetics. Part A, 2020, v. 182, n. 5, p. 1053, doi. 10.1002/ajmg.a.61518
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- Publication type:
- Article
Immune-Mediated Inflammation May Contribute to the Pathogenesis of Cardiovascular Disease in Mucopolysaccharidosis Type I.
- Published in:
- PLoS ONE, 2016, v. 11, n. 3, p. 1, doi. 10.1371/journal.pone.0150850
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- Publication type:
- Article