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Journal Article
Journal of investigative dermatology, ISSN 0022-202X, 11/2016, Volume 136, Issue 11, pp. 2119 - 2121
Epidermolysis bullosa is a group of heritable skin fragility disorders with considerable morbidity and mortality... 
Life Sciences & Biomedicine | Dermatology | Science & Technology | Epidermolysis Bullosa - therapy | Epidermolysis Bullosa - genetics | Stem Cell Transplantation - methods | Humans | Genetic Therapy - methods | Index Medicus
Journal Article
Journal Article
Molecular therapy, ISSN 1525-0016, 11/2017, Volume 25, Issue 11, pp. 2573 - 2584
... blistering skin disease recessive dystrophic epidermolysis bullosa. Upon CRISPR/Cas9 treatment of patient-derived keratinocytes, using either the wild-type Cas9 or D10A... 
CRISPR/Cas9 | homology-directed repair | epidermolysis bullosa | COL7A1 | Genetics & Heredity | Life Sciences & Biomedicine | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Exons | Humans | Transplantation, Heterologous | Molecular Targeted Therapy | Epidermolysis Bullosa Dystrophica - pathology | Base Sequence | Keratinocytes - transplantation | Collagen Type VII - metabolism | Epidermolysis Bullosa Dystrophica - metabolism | Epidermolysis Bullosa Dystrophica - therapy | Gene Expression | Collagen Type VII - genetics | Treatment Outcome | Plasmids - metabolism | Keratinocytes - pathology | Animals | Keratinocytes - metabolism | Mice, Nude | CRISPR-Cas Systems | Plasmids - chemistry | Gene Editing - methods | High-Throughput Nucleotide Sequencing | Mice | Epidermolysis Bullosa Dystrophica - genetics | Mutation | Primary Cell Culture | Disease | Homology | Transplantation | Genomes | Proteins | Design | Reversion | Next-generation sequencing | Efficiency | Phenotypic reversion | Skin diseases | Nuclease | Localization | Deoxyribonucleic acid--DNA | CRISPR | Epidermolysis bullosa | Cloning | Immunodeficiency | Keratinocytes | Vectors (Biology) | Microscopy | Collagen | Skin | Gene therapy | Binding sites | Dystrophic epidermolysis bullosa | Index Medicus | Cas9 | Original
Journal Article