Gene activation of SMN by selective disruption of lncRNA-mediated recruitment of PRC2 for the treatment of spinal muscular atrophy

Proceedings of the National Academy of Sciences of the United States of America

Volumn 114, Issue 8, 2017, Pages E1509-E1518

  Woo, Caroline J ^a   Maier, Verena K ^a   Davey, Roshni ^a   Brennan, James ^a   Li, Guangde ^a   Brothers, John ^a   Schwartz, Brian ^a   Gordo, Susana ^a   Kasper, Anne ^a   Okamoto, Trevor R ^b   Johansson, Hans E ^b   Mandefro, Berhan ^c   Sareen, Dhruv ^c   Bialek, Peter ^a   Chau, B Nelson ^a   Bhat, Balkrishen ^a   Bullough, David ^a   Barsoum, James ^a  

^a Translate Bio Inc   (United States)

^b LGC   (United Kingdom)

^c CEDARS SINAI MEDICAL CENTER   (United States)

Author keywords

LncRNA; PRC2; SMN; Spinal muscular atrophy

Indexed keywords

LONG UNTRANSLATED RNA; MESSENGER RNA; OLIGONUCLEOTIDE; POLYCOMB REPRESSIVE COMPLEX 2; SURVIVAL MOTOR NEURON PROTEIN 2; SMN1 PROTEIN, HUMAN; SMN1 PROTEIN, MOUSE; SMN2 PROTEIN, HUMAN; SMN2 PROTEIN, MOUSE; SURVIVAL MOTOR NEURON PROTEIN 1;

ADULT; ARTICLE; BASE PAIRING; CELL STRUCTURE; CHROMATIN IMMUNOPRECIPITATION; DISSOCIATION CONSTANT; ENZYME LINKED IMMUNOSORBENT ASSAY; FETUS; FLUORESCENCE IN SITU HYBRIDIZATION; GEL MOBILITY SHIFT ASSAY; GENE ACTIVATION; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; HUMAN TISSUE; IMMUNOPRECIPITATION; INDUCED PLURIPOTENT STEM CELL; NERVE CELL CULTURE; NORTHERN BLOTTING; POLYMERASE CHAIN REACTION SYSTEM; PRIORITY JOURNAL; PROTEIN EXPRESSION; QUANTITATIVE ANALYSIS; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; RNA SEQUENCE; SPINAL MUSCULAR ATROPHY; TRANSCRIPTION INITIATION; TRANSCRIPTION REGULATION; UPREGULATION; WESTERN BLOTTING; ANIMAL; CELL LINE; DISEASE MODEL; EXON; FIBROBLAST; GENE DOSAGE; GENE THERAPY; GENETICS; METABOLISM; MOLECULARLY TARGETED THERAPY; MOTONEURON; MOUSE; POINT MUTATION; PROCEDURES;

ANIMALS; CELL LINE; DISEASE MODELS, ANIMAL; EXONS; FIBROBLASTS; GENE DOSAGE; GENETIC THERAPY; HUMANS; MICE; MOLECULAR TARGETED THERAPY; MOTOR NEURONS; MUSCULAR ATROPHY, SPINAL; OLIGONUCLEOTIDES; POINT MUTATION; POLYCOMB REPRESSIVE COMPLEX 2; RNA, LONG NONCODING; SURVIVAL OF MOTOR NEURON 1 PROTEIN; SURVIVAL OF MOTOR NEURON 2 PROTEIN; TRANSCRIPTIONAL ACTIVATION; UP-REGULATION;

EID: 85013249596     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.1616521114     Document Type: Article

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