Gene therapy in spinal muscular atrophy (SMA) models using intracerebroventricular injection into neonatal mice

Neuromethods

Volumn 98, Issue , 2015, Pages 297-320

  Shababi, Monir ^a   Osman, Erkan Y ^a   Lorson, Christian L ^a  

^a NONE

Author keywords

AAV vectors; Gene therapy; ICV injection; SMA; SMN

Indexed keywords

PARVOVIRUS VECTOR; SURVIVAL MOTOR NEURON PROTEIN;

ADENO ASSOCIATED VIRUS; ARTICLE; CENTRAL NERVOUS SYSTEM; GENE REPLACEMENT THERAPY; GENE THERAPY; HUMAN; NONHUMAN; PERINATAL PERIOD; PHASE 1 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; RNA SPLICING; SPINAL MUSCULAR ATROPHY; TRANSGENE; VIRAL GENE DELIVERY SYSTEM;

ANIMALIA; MURINAE; MUS;

EID: 84961342221     PISSN: 08932336     EISSN: 19406045     Source Type: Book Series    
DOI: 10.1007/978-1-4939-2306-9_12     Document Type: Article

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