Survival benefit and phenotypic improvement by hamartin gene therapy in a tuberous sclerosis mouse brain model

Neurobiology of Disease

Volumn 82, Issue , 2015, Pages 22-31

  Prabhakar, Shilpa ^a   Zhang, Xuan ^a   Goto, June ^b   Han, Sangyeul ^a   Lai, Charles ^a   Bronson, Roderick ^c   Sena Esteves, Miguel ^d   Ramesh, Vijaya ^a   Stemmer Rachamimov, Anat ^a   Kwiatkowski, David J ^b   Breakefield, Xandra O ^a  

^a MASSACHUSETTS GENERAL HOSPITAL   (United States)

^b BRIGHAM AND WOMEN S HOSPITAL   (United States)

^c HARVARD MEDICAL SCHOOL   (United States)

^d UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

Author keywords

AAV; Gene therapy; Neuron; TSC; TSC1; TSC2; Tuberous sclerosis complex

Indexed keywords

HAMARTIN; MAMMALIAN TARGET OF RAPAMYCIN; PARVOVIRUS VECTOR; TUBEROUS SCLEROSIS COMPLEX 1 PROTEIN; TUMOR SUPPRESSOR PROTEIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; GENE EXPRESSION; GENE THERAPY; GENOTYPE; HISTOPATHOLOGY; HUMAN; HUMAN CELL; LOCOMOTION; MOUSE; NEWBORN; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; SURVIVAL; TUBEROUS SCLEROSIS; TUBEROUS SCLEROSIS COMPLEX; VIRAL GENE DELIVERY SYSTEM; WEIGHT GAIN; ANIMAL; BRAIN; DISEASE MODEL; GENETICS; MUTATION; NERVE CELL; PATHOLOGY; PROCEDURES; TREATMENT OUTCOME;

ANIMALS; BRAIN; DISEASE MODELS, ANIMAL; GENETIC THERAPY; MICE; MUTATION; NEURONS; PHENOTYPE; TREATMENT OUTCOME; TUBEROUS SCLEROSIS; TUMOR SUPPRESSOR PROTEINS;

EID: 84930960323     PISSN: 09699961     EISSN: 1095953X     Source Type: Journal    
DOI: 10.1016/j.nbd.2015.04.018     Document Type: Article

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