Genome Therapy of Myotonic Dystrophy Type 1 iPS Cells for Development of Autologous Stem Cell Therapy. [electronic resource]
Producer: 20170606Description: 1378-87 p. digitalISSN:- 1525-0024
- Animals
- Cell Differentiation
- Cell Nucleus -- metabolism
- Cell Transformation, Neoplastic -- genetics
- Disease Models, Animal
- Gene Targeting
- Genetic Loci
- Genetic Therapy
- Humans
- Induced Pluripotent Stem Cells -- cytology
- Mice
- Myocytes, Cardiac -- cytology
- Myotonic Dystrophy -- genetics
- Myotonin-Protein Kinase -- genetics
- Poly A
- Protein Binding
- RNA Splicing
- Stem Cell Transplantation
- Teratoma -- genetics
- Transcription Activator-Like Effector Nucleases
- Transplantation, Autologous
- Trinucleotide Repeats
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Publication Type: Journal Article; Research Support, Non-U.S. Gov't; Research Support, N.I.H., Extramural
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