Romero-Moya, Damià

Genetic Rescue of Mitochondrial and Skeletal Muscle Impairment in an Induced Pluripotent Stem Cells Model of Coenzyme Q [electronic resource] - Stem cells (Dayton, Ohio) 07 2017 - 1687-1703 p. digital

Publication Type: Case Reports; Journal Article; Research Support, Non-U.S. Gov't

1549-4918

10.1002/stem.2634 doi


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