Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy

Molecular Therapy Methods and Clinical Development

Volumn 3, Issue , 2016, Pages 16060-

  Armbruster, Nicole ^a,b   Lattanzi, Annalisa ^a,b   Jeavons, Matthieu ^a,b   Van Wittenberghe, Laetitia ^b   Gjata, Bernard ^b   Marais, Thibaut ^c   Martin, Samia ^b   Vignaud, Alban ^b   Voit, Thomas ^c   Mavilio, Fulvio ^a,b   Barkats, Martine ^c   Buj Bello, Ana ^a,b  

^a INSERM UMR 951 ^*  (France)

^b GÉNÉTHON   (France)

^c CNRS   (France)

Author keywords

[No Author keywords available]

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; PHOSPHOGLYCERATE KINASE; SURVIVAL MOTOR NEURON PROTEIN 1; SURVIVAL MOTOR NEURON PROTEIN;

ANIMAL CELL; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CEREBROSPINAL FLUID; CODON; CONTROLLED STUDY; DRUG DOSE INCREASE; IMMUNOHISTOCHEMISTRY; LUMBAR SPINAL CORD; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROMOTER REGION; PROTEIN EXPRESSION; SPINAL MUSCULAR ATROPHY; SURVIVAL; THERAPY EFFECT; VIRAL GENE THERAPY; ANIMAL EXPERIMENT; DOSE RESPONSE; GENE EXPRESSION; GENETIC CODE; HEART; LIFESPAN; LIVER; NEWBORN; PHENOTYPE; POST TREATMENT SURVIVAL; PROTEIN SYNTHESIS; SKELETAL MUSCLE; SPINAL CORD; VIRAL GENE DELIVERY SYSTEM;

EID: 85015253691     PISSN: None     EISSN: 23290501     Source Type: Journal    
DOI: 10.1038/mtm.2016.60     Document Type: Article

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