Upregulation of Haploinsufficient Gene Expression in the Brain by Targeting a Long Non-coding RNA Improves Seizure Phenotype in a Model of Dravet Syndrome

EBioMedicine

Volumn 9, Issue , 2016, Pages 257-277

  Hsiao, J ^a   Yuan, T Y ^a   Tsai, M S ^b   Lu, C Y ^c   Lin, Y C ^a   Lee, M L ^b   Lin, S W ^b,c   Chang, F C ^c,d,e   Liu Pimentel, H ^a   Olive, C ^a   Coito, C ^a   Shen, G ^a   Young, M ^a   Thorne, T ^a   Lawrence, M ^f   Magistri, M ^g   Faghihi, M A ^g   Khorkova, O ^a   Wahlestedt, C ^g  

^a OPKO Health Inc   (United States)

^b NATIONAL TAIWAN UNIVERSITY HOSPITAL   (Taiwan)

^c NATIONAL TAIWAN UNIVERSITY   (Taiwan)

^d NATIONAL TAIWAN UNIVERSITY   (Taiwan)

^e CHINA MEDICAL UNIVERSITY   (Taiwan)

^f RxGen Inc   (United States)

^g UNIVERSITY OF MIAMI MILLER SCHOOL OF MEDICINE   (United States)

Author keywords

AntagoNAT; Dravet syndrome; Long non coding RNA; Natural antisense transcript; Oligonucleotide based compound; SCN1A

Indexed keywords

ANIMAL MODEL; ARTICLE; ELECTROENCEPHALOGRAPHY; GENE EXPRESSION; GENE SEQUENCE; GENETIC DISORDER; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; HUMAN TISSUE; IMMUNOHISTOCHEMISTRY; MOLECULAR CLONING; MOUSE; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; REAL TIME POLYMERASE CHAIN REACTION; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; SEIZURE; SEVERE MYOCLONIC EPILEPSY IN INFANCY; SKIN BIOPSY; UPREGULATION; ALLELE; ANIMAL; ANIMAL BEHAVIOR; ANTAGONISTS AND INHIBITORS; BRAIN; CELL LINE; CHEMISTRY; CHLOROCEBUS AETHIOPS; CONFORMATION; DIAGNOSTIC IMAGING; DISEASE MODEL; GENE TARGETING; GENETICS; HIPPOCAMPUS; IN VITRO STUDY; INTERNEURON; METABOLISM; MOLECULAR GENETICS; MYOCLONUS EPILEPSY; NUCLEOTIDE SEQUENCE; PATCH CLAMP TECHNIQUE; PATHOLOGY; PHYSIOLOGY; SEQUENCE ALIGNMENT; SEQUENCE ANALYSIS; TEMPERATURE; TRANSGENIC MOUSE; VERO CELL LINE; VIDEORECORDING;

ANTISENSE OLIGONUCLEOTIDE; LONG UNTRANSLATED RNA; SODIUM CHANNEL NAV1.1;

ALLELES; ANIMALS; BASE SEQUENCE; BEHAVIOR, ANIMAL; BRAIN; CELL LINE; CERCOPITHECUS AETHIOPS; DISEASE MODELS, ANIMAL; ELECTROENCEPHALOGRAPHY; EPILEPSIES, MYOCLONIC; GENE EXPRESSION; GENE KNOCK-IN TECHNIQUES; HIPPOCAMPUS; HUMANS; IN VITRO TECHNIQUES; INTERNEURONS; MICE; MICE, TRANSGENIC; MOLECULAR SEQUENCE DATA; NAV1.1 VOLTAGE-GATED SODIUM CHANNEL; NUCLEIC ACID CONFORMATION; OLIGONUCLEOTIDES, ANTISENSE; PATCH-CLAMP TECHNIQUES; PHENOTYPE; REAL-TIME POLYMERASE CHAIN REACTION; RNA, LONG NONCODING; SEQUENCE ALIGNMENT; SEQUENCE ANALYSIS, RNA; TEMPERATURE; UP-REGULATION; VERO CELLS; VIDEO RECORDING;

EID: 84970044611     PISSN: None     EISSN: 23523964     Source Type: Journal    
DOI: 10.1016/j.ebiom.2016.05.011     Document Type: Article

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