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Molecular Genetics and Metabolism, ISSN 1096-7192, 01/2017, Volume 120, Issue 1-2, pp. S40 - S40
Journal Article
Biotechnology Letters, ISSN 0141-5492, 3/2018, Volume 40, Issue 3, pp. 617 - 622
Mesenchymal stromal cells (MSCs) are potential targets for cell and gene therapy-based approaches against a variety of different diseases. The MSCs from bone... 
Life Sciences | Biochemistry, general | Mesenchymal stem cell | Biotechnology | Transfection | Microbiology | Applied Microbiology | Gene therapy | THERAPY | GENE DELIVERY | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | EXPANSION | EFFICIENCY | VECTORS | Genetic research | Research | Hematopoietic stem cells | Mesenchyme | Stromal cells | Stem cells | Bone marrow | Deoxyribonucleic acid--DNA
Journal Article
European Journal of Pharmaceutics and Biopharmaceutics, ISSN 0939-6411, 01/2018, Volume 122, pp. 158 - 166
Mucopolysaccharidosis type I (MPS I) is an inherited disease caused by the deficiency of alpha-L-iduronidase (IDUA). This study shows the use of nanoemulsions... 
Mucopolysaccharidosis type I | Nonviral vectors | CRISPR/Cas9 | Gene therapy | Gene editing | Nanoemulsion | MUCOPOLYSACCHARIDOSIS TYPE-I | SYSTEM | TRANSFECTION | COMPLEXES | LIPID-BASED EMULSION | Nenoemulsion | MURINE MODEL | THERAPY | PHARMACOLOGY & PHARMACY | CATIONIC NANOEMULSIONS | MUTATIONS | VECTORS | Adsorption | Genes
Journal Article
Pharmaceutical Research, ISSN 0724-8741, 3/2015, Volume 32, Issue 3, pp. 941 - 954
Journal Article
Gene, ISSN 0378-1119, 12/2018, Volume 678, pp. 33 - 37
Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disorder (LSD). It is caused by mutations in the gene, which lead to the accumulation of the... 
Hurler syndrome | Mucopolysaccharidosis | Gene editing | CRISPR-Cas9 | MUCOPOLYSACCHARIDOSIS TYPE-I | DISEASE | GENETICS & HEREDITY | MUTATIONS | IDENTIFICATION | Enzymes | Anopheles | Analysis | Genes | Genomics | Stem cells | Genetic research | Sulfates
Journal Article
PLoS ONE, ISSN 1932-6203, 02/2015, Volume 10, Issue 2, p. e0117271
Journal Article
Journal Article
Journal of Controlled Release, ISSN 0168-3659, 07/2015, Volume 209, pp. 37 - 46
Journal Article
Current Stem Cell Reports, ISSN 2198-7866, 3/2015, Volume 1, Issue 1, pp. 9 - 15
The recent progress in genome editing technology using the engineered zinc finger nucleases (ZFN), transcriptional activator-like effector nucleases (TALEN),... 
Life Sciences | Stem Cells | TAL effector nucleases | Lysosomal storage disorders | CRISPR-Cas9 | Zinc finger nucleases | Cell Biology
Journal Article
Journal Article