Found: 15
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Identification of Orthosteric and Allosteric Pharmacological Chaperones for Mucopolysaccharidosis Type IIIB.
- Published in:
- ChemBioChem, 2024, v. 25, n. 15, p. 1, doi. 10.1002/cbic.202400081
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- Publication type:
- Article
Study of Potential Blocking Peptides Targeting the SARS-CoV-2 RBD/hACE2 Interaction.
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- Pharmaceuticals (14248247), 2024, v. 17, n. 9, p. 1240, doi. 10.3390/ph17091240
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- Publication type:
- Article
CRISPR/nCas9-Based Genome Editing on GM2 Gangliosidoses Fibroblasts via Non-Viral Vectors.
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- International Journal of Molecular Sciences, 2022, v. 23, n. 18, p. N.PAG, doi. 10.3390/ijms231810672
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- Article
GM2 Gangliosidoses: Clinical Features, Pathophysiological Aspects, and Current Therapies.
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- International Journal of Molecular Sciences, 2020, v. 21, n. 17, p. 6213, doi. 10.3390/ijms21176213
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- Publication type:
- Article
Delivery and assessment of a CRISPR/nCas9-based genome editing system on in vitro models of mucopolysaccharidoses IVA assisted by magnetite-based nanoparticles.
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- Scientific Reports, 2022, v. 12, n. 1, p. 1, doi. 10.1038/s41598-022-19407-x
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- Publication type:
- Article
Human recombinant lysosomal β‐Hexosaminidases produced in Pichia pastoris efficiently reduced lipid accumulation in Tay‐Sachs fibroblasts.
- Published in:
- American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2020, v. 184, n. 4, p. 885, doi. 10.1002/ajmg.c.31849
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- Article
Recombinant human N-acetylgalactosamine-6-sulfate sulfatase (GALNS) produced in the methylotrophic yeast Pichia pastoris.
- Published in:
- Scientific Reports, 2016, p. 29329, doi. 10.1038/srep29329
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- Publication type:
- Article
Therapeutic Options for Mucopolysaccharidoses: Current and Emerging Treatments.
- Published in:
- Drugs, 2019, v. 79, n. 10, p. 1103, doi. 10.1007/s40265-019-01147-4
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- Publication type:
- Article
Neural stem cells for disease modeling and evaluation of therapeutics for Tay-Sachs disease.
- Published in:
- 2018
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- Publication type:
- journal article
Research, diagnosis and education in inborn errors of metabolism in Colombia: 20 years' experience from a reference center.
- Published in:
- 2018
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- Publication type:
- journal article
impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome.
- Published in:
- Drug Design, Development & Therapy, 2015, v. 9, p. 1937, doi. 10.2147/DDDT.S68562
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- Publication type:
- Article
Characterization and expression of domains of Alphaherpesvirus bovine 1/5 envelope glycoproteins B in Komagataella phaffi.
- Published in:
- BMC Veterinary Research, 2023, v. 19, n. 1, p. 1, doi. 10.1186/s12917-023-03590-8
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- Publication type:
- Article
Production and characterization of a human lysosomal recombinant iduronate‐2‐sulfatase produced in Pichia pastoris.
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- Biotechnology & Applied Biochemistry, 2018, v. 65, n. 5, p. 655, doi. 10.1002/bab.1660
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- Publication type:
- Article
Production of Recombinant Trichoderma reesei Cellobiohydrolase II in a New Expression System Based on Wickerhamomyces anomalus.
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- Enzyme Research, 2017, p. 1, doi. 10.1155/2017/6980565
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- Publication type:
- Article
Lysosomal storage diseases: current therapies and future alternatives.
- Published in:
- Journal of Molecular Medicine, 2020, v. 98, n. 7, p. 931, doi. 10.1007/s00109-020-01935-6
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- Publication type:
- Article