Development of Therapeutics for C9ORF72 ALS/FTD-Related Disorders

Molecular Neurobiology

Volumn 54, Issue 6, 2017, Pages 4466-4476

  Mis, Maria Sara Cipolat ^a   Brajkovic, Simona ^a   Tafuri, Francesco ^a   Bresolin, Nereo ^a   Comi, Giacomo P ^a   Corti, Stefania ^a  

^a UNIVERSITY OF MILAN   (Italy)

Author keywords

Antisense oligonucleotides; Haploinsufficiency; Hexanucleotide repeat expansion

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; CHROMOSOME 9 OPEN READING FRAME 72 PROTEIN; CHROMOSOME PROTEIN; RNA; RNA BINDING PROTEIN; UNCLASSIFIED DRUG; GUANINE NUCLEOTIDE EXCHANGE C9ORF72;

AMYOTROPHIC LATERAL SCLEROSIS; CELL NUCLEUS; CYTOPLASM; FRONTOTEMPORAL DEMENTIA; GENE EXPRESSION; GENE SILENCING; GENE THERAPY; GENETIC TRANSCRIPTION; HAPLOINSUFFICIENCY; HEXANUCLEOTIDE REPEAT EXPANSION; HUMAN; NONHUMAN; NUCLEOTIDE REPEAT; PATHOGENESIS; REVIEW; RNA INTERFERENCE; RNA PROCESSING; RNA SEQUENCE; ANIMAL; GENETICS;

AMYOTROPHIC LATERAL SCLEROSIS; ANIMALS; C9ORF72 PROTEIN; FRONTOTEMPORAL DEMENTIA; GENETIC THERAPY; HUMANS; OLIGONUCLEOTIDES, ANTISENSE; RNA INTERFERENCE;

EID: 84976317472     PISSN: 08937648     EISSN: 15591182     Source Type: Journal    
DOI: 10.1007/s12035-016-9993-0     Document Type: Review

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