Rescue of amyotrophic lateral sclerosis phenotype in a mouse model by intravenous AAV9-ADAR2 delivery to motor neurons

EMBO Molecular Medicine

Volumn 5, Issue 11, 2013, Pages 1710-1719

  Yamashita, Takenari ^a,b   Chai, Hui Lin ^a,b   Teramoto, Sayaka ^a,b   Tsuji, Shoji ^b   Shimazaki, Kuniko ^c   Muramatsu, Shin ichi ^c   Kwak, Shin ^a,b,d  

^a JAPAN SCIENCE AND TECHNOLOGY AGENCY   (Japan)

^b UNIVERSITY OF TOKYO   (Japan)

^c JICHI MEDICAL UNIVERSITY   (Japan)

^d INTERNATIONAL UNIVERSITY OF HEALTH AND WELFARE   (Japan)

Author keywords

Adeno associated virus (AAV) 9; Adenosine deaminase acting on RNA 2 (ADAR2); AMPA receptor; Amyotrophic lateral sclerosis (ALS); Gene therapy

Indexed keywords

ADENOSINE DEAMINASE; AMPA RECEPTOR; CALCIUM; PARVOVIRUS VECTOR; PROTEIN ADAR2; TAR DNA BINDING PROTEIN; UNCLASSIFIED DRUG;

ADENO ASSOCIATED VIRUS; ADENO ASSOCIATED VIRUS 9; AMYOTROPHIC LATERAL SCLEROSIS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DOWN REGULATION; DRUG EFFICACY; DRUG MECHANISM; KNOCKOUT MOUSE; MOTONEURON; MOTOR DYSFUNCTION; MOUSE; NERVE CELL NECROSIS; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; RNA EDITING; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

ADENO-ASSOCIATED VIRUS (AAV) 9; ADENOSINE DEAMINASE ACTING ON RNA 2 (ADAR2); AMPA RECEPTOR; AMYOTROPHIC LATERAL SCLEROSIS (ALS); GENE THERAPY;

ADENOSINE DEAMINASE; AMYOTROPHIC LATERAL SCLEROSIS; ANIMALS; BRAIN; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MICE; MICE, KNOCKOUT; MICE, TRANSGENIC; MOTOR NEURONS; RNA-BINDING PROTEINS;

EID: 84887084505     PISSN: 17574676     EISSN: 17574684     Source Type: Journal    
DOI: 10.1002/emmm.201302935     Document Type: Article

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