Rescue of a mouse model of spinal muscular atrophy with respiratory distress type 1 by AAV9-IGHMBP2 is dose dependent

Molecular Therapy

Volumn 24, Issue 5, 2016, Pages 855-866

  Shababi, Monir ^a,b   Feng, Zhihua ^c   Villalon, Eric ^b   Sibigtroth, Christine M ^b   Osman, Erkan Y ^a,b   Miller, Madeline R ^b   Williams Simon, Patricka A ^b   Lombardi, Abby ^b   Sass, Thalia H ^b   Atkinson, Arleigh K ^b   Garcia, Michael L ^b   Ko, Chien Ping ^c   Lorson, Christian L ^a,b  

^a University of Missouri   (United States)

^b UNIVERSITY OF MISSOURI   (United States)

^c UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BINDING PROTEIN; COMPLEMENTARY DNA; IMMUNOGLOBULIN MU BINDING PROTEIN 2; PARVOVIRUS VECTOR; UNCLASSIFIED DRUG; DNA BINDING PROTEIN; IGHMBP2 PROTEIN, HUMAN; TRANSCRIPTION FACTOR;

ADENO ASSOCIATED VIRUS 9; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DOSE RESPONSE; DRUG DOSE COMPARISON; DRUG EFFICACY; DRUG MEGADOSE; GENE EXPRESSION REGULATION; HINDLIMB; IGHMBP2 GENE; IN VIVO STUDY; LOW DRUG DOSE; LUMBAR SPINAL CORD; MALE; MOTOR DYSFUNCTION; MOUSE; MOUSE MODEL; MUSCLE DENERVATION; NONHUMAN; PHENOTYPE; RESPIRATORY DISTRESS; SPINAL CORD VENTRAL HORN; SPINAL MUSCULAR ATROPHY; SPINAL MUSCULAR ATROPHY WITH RESPIRATORY DISTRESS TYPE 1; VIRAL GENE DELIVERY SYSTEM; ADMINISTRATION AND DOSAGE; ANIMAL; BODY WEIGHT; DEPENDOPARVOVIRUS; DISEASE MODEL; FEMALE; GENE THERAPY; GENE VECTOR; GENETICS; HUMAN; MUSCULAR ATROPHY, SPINAL; MUTATION; RESPIRATORY DISTRESS SYNDROME, NEWBORN; SURVIVAL ANALYSIS;

ANIMALS; BODY WEIGHT; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DNA-BINDING PROTEINS; DOSE-RESPONSE RELATIONSHIP, DRUG; FEMALE; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MALE; MICE; MUSCULAR ATROPHY, SPINAL; MUTATION; RESPIRATORY DISTRESS SYNDROME, NEWBORN; SURVIVAL ANALYSIS; TRANSCRIPTION FACTORS;

EID: 84960192546     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2016.33     Document Type: Article

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