Targeting RNA foci in iPSC-derived motor neurons from ALS patients with a C9ORF72 repeat expansion

Science Translational Medicine

Volumn 5, Issue 208, 2013, Pages

  Sareen, Dhruv ^a   O'Rourke, Jacqueline G ^a   Meera, Pratap ^b   Muhammad, A K M G ^a   Grant, Sharday ^a   Simpkinson, Megan ^a   Bell, Shaughn ^a   Carmona, Sharon ^a   Ornelas, Loren ^a   Sahabian, Anais ^a   Gendron, Tania ^c   Petrucelli, Leonard ^c   Baughn, Michael ^d   Ravits, John ^d   Harms, Matthew B ^e   Rigo, Frank ^f   Frank Bennett, C ^f   Otis, Thomas S ^b   Svendsen, Clive N ^a   Baloh, Robert H ^a  

^a CEDARS SINAI MEDICAL CENTER   (United States)

^b UNIVERSITY OF CALIFORNIA   (United States)

^c MAYO CLINIC   (United States)

^d UNIVERSITY OF CALIFORNIA   (United States)

^e WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^f ISIS PHARMACEUTICALS   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

POTASSIUM CHANNEL KCNQ3; RNA BINDING PROTEIN;

AMYOTROPHIC LATERAL SCLEROSIS; ARTICLE; C9ORF72 GENE; CELL DIFFERENTIATION; CONTROLLED STUDY; DISEASE SEVERITY; DNA BASE COMPOSITION; DOWN REGULATION; EXON; FIBROBLAST; GENE; GENE EXPRESSION; GENE FREQUENCY; GENE FUNCTION; GENE LOCUS; GENE TARGETING; HUMAN; HUMAN CELL; KARYOTYPE; MOTONEURON; PHENOTYPE; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; RNA METABOLISM; RNA SPLICING; SINGLE NUCLEOTIDE POLYMORPHISM;

AMYOTROPHIC LATERAL SCLEROSIS; DNA REPEAT EXPANSION; EXONS; GENE KNOCKDOWN TECHNIQUES; HUMANS; INDUCED PLURIPOTENT STEM CELLS; MOTOR NEURONS; OLIGONUCLEOTIDES, ANTISENSE; PROTEINS; RNA; TRANSCRIPTION, GENETIC;

EID: 84886389563     PISSN: 19466234     EISSN: 19466242     Source Type: Journal    
DOI: 10.1126/scitranslmed.3007529     Document Type: Article

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