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JAMA, ISSN 0098-7484, 11/2016, Volume 316, Issue 17, pp. 1808 - 1817
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 08/2010, Volume 363, Issue 7, pp. 629 - 639
Journal Article
Journal Article
American Journal of Pathology, The, ISSN 0002-9440, 2017, Volume 187, Issue 7, pp. 1445 - 1453
Journal Article
Molecular Therapy, ISSN 1525-0016, 11/2017, Volume 25, Issue 11, pp. 2573 - 2584
Designer nucleases allow specific and precise genomic modifications and represent versatile molecular tools for the correction of disease-associated mutations.... 
CRISPR/Cas9 | homology-directed repair | epidermolysis bullosa | COL7A1 | MEDICINE, RESEARCH & EXPERIMENTAL | CRISPR-CAS9 | MINICIRCLE DNA | MECHANISMS | TRANSPLANTATION | PLURIPOTENT STEM-CELLS | REPAIR | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | IN-VIVO | MOUSE MODEL | DISEASE | GENETICS & HEREDITY | GENE-THERAPY | 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 | Cas9 | Original
Journal Article
Neuron, ISSN 0896-6273, 06/2013, Volume 78, Issue 5, pp. 785 - 798
Available methods for differentiating human embryonic stem cells (ESCs) and induced pluripotent cells (iPSCs) into neurons are often cumbersome, slow, and... 
HUMAN ES | RETINOIC ACID | DISEASE | DIRECT CONVERSION | HUMAN FIBROBLASTS | HOMEOSTATIC SYNAPTIC PLASTICITY | DOPAMINERGIC-NEURONS | DIFFERENTIATION | NEUROSCIENCES | HIGHLY EFFICIENT | IPS CELLS | Electric Stimulation | Calcium - metabolism | Humans | Munc18 Proteins - metabolism | Green Fluorescent Proteins - genetics | Excitatory Postsynaptic Potentials - drug effects | Epidermolysis Bullosa Dystrophica - pathology | Transfection | Time Factors | Neurons - physiology | 6-Cyano-7-nitroquinoxaline-2,3-dione - pharmacology | Fibroblasts | Biophysical Phenomena - genetics | Tetrodotoxin - pharmacology | Excitatory Postsynaptic Potentials - genetics | Brain - cytology | Munc18 Proteins - genetics | Gene Expression Regulation - genetics | Synapses - physiology | Pluripotent Stem Cells - physiology | Cells, Cultured | Gene Expression Regulation - physiology | Collagen Type VII - genetics | RNA, Small Interfering - physiology | Sodium Channel Blockers - pharmacology | Excitatory Amino Acid Antagonists - pharmacology | Biophysics | Mutation - genetics | Nerve Tissue Proteins - genetics | Biophysical Phenomena - physiology | Gene Expression Regulation - drug effects | Nerve Tissue Proteins - metabolism | Microscopy, Confocal | Patch-Clamp Techniques | Animals | Rhodopsin - genetics | Mice | Epidermolysis Bullosa Dystrophica - genetics | Medical colleges | Embryonic stem cells | Neurons | Analysis | Studies | Transcription factors | Alzheimers disease | Experiments | Stem cells
Journal Article