Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy

Human Gene Therapy

Volumn 25, Issue 7, 2014, Pages 619-630

  Passini, Marco A ^a   Bu, Jie ^a   Richards, Amy M ^a   Treleaven, Christopher M ^a   Sullivan, Jennifer A ^a   O'Riordan, Catherine R ^a   Scaria, Abraham ^a   Kells, Adrian P ^b   Samaranch, Lluis ^b   San Sebastian, Waldy ^b   Federici, Thais ^c   Fiandaca, Massimo S ^d   Boulis, Nicholas M ^c   Bankiewicz, Krystof S ^b   Shihabuddin, Lamya S ^a   Cheng, Seng H ^a  

^a SANOFI   (France)

^b UNIVERSITY OF CALIFORNIA   (United States)

^c EMORY UNIVERSITY SCHOOL OF MEDICINE   (United States)

^d GEORGETOWN UNIVERSITY MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

SELF COMPLEMENTARY AAV9 SURVIVAL MOTOR NEURON PROTEIN 1; SURVIVAL MOTOR NEURON PROTEIN; UNCLASSIFIED DRUG; SMN1 PROTEIN, MOUSE; SURVIVAL MOTOR NEURON PROTEIN 1;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL COUNT; CONTROLLED STUDY; DOSE RESPONSE; FEMALE; GENE DOSAGE; GENE EXPRESSION; GENE THERAPY; MALE; MOTONEURON; MOUSE; NONHUMAN; SPINAL MUSCULAR ATROPHY; SURVIVAL RATE; SWINE; ANIMAL; BIOSYNTHESIS; DEPENDOPARVOVIRUS; GENE VECTOR; GENETICS; INTRASPINAL DRUG ADMINISTRATION; KNOCKOUT MOUSE; METABOLISM; MUSCULAR ATROPHY, SPINAL; PATHOLOGY; PHARMACOLOGY; PROTEIN SYNTHESIS; SPINAL CORD;

ANIMALS; DEPENDOVIRUS; GENETIC VECTORS; INJECTIONS, SPINAL; MICE; MICE, KNOCKOUT; MOTOR NEURONS; MUSCULAR ATROPHY, SPINAL; PROTEIN BIOSYNTHESIS; SPINAL CORD; SURVIVAL OF MOTOR NEURON 1 PROTEIN; SWINE;

EID: 84904412918     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2014.011     Document Type: Article

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