Genetic Rescue of Mitochondrial and Skeletal Muscle Impairment in an Induced Pluripotent Stem Cells Model of Coenzyme Q [electronic resource]
Producer: 20180323Description: 1687-1703 p. digitalISSN:- 1549-4918
- Ataxia -- enzymology
- CRISPR-Cas Systems
- Cell Differentiation
- Child, Preschool
- Dopaminergic Neurons -- cytology
- Electron Transport Chain Complex Proteins -- genetics
- Fatal Outcome
- Female
- Fibroblasts -- metabolism
- Gene Editing -- methods
- Gene Expression
- Genes, Lethal
- Humans
- Induced Pluripotent Stem Cells -- metabolism
- Intellectual Disability -- enzymology
- Mitochondria -- enzymology
- Mitochondrial Diseases -- enzymology
- Mitochondrial Proteins -- deficiency
- Motor Neurons -- cytology
- Muscle Weakness -- enzymology
- Primary Cell Culture
- Rhabdomyolysis -- enzymology
- Ubiquinone -- analogs & derivatives
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Publication Type: Case Reports; Journal Article; Research Support, Non-U.S. Gov't
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