Intravitreal injection of splice-switching oligonucleotides to manipulate splicing in retinal cells

Molecular Therapy Nucleic Acids

Volumn 4, Issue , 2015, Pages e250-

  Gérard, Xavier ^a   Perrault, Isabelle ^a   Munnich, Arnold ^a   Kaplan, Josseline ^a   Rozet, Jean Michel ^a  

^a IMAGINE INSTITUTE   (France)

Author keywords

2 OMePS antisense oligonucleotide; CEP290; intravitreal injection; Leber congenital amaurosis; retinal dystrophies; splice switching therapy

Indexed keywords

ABCA4 PROTEIN; MEMBRANE PROTEIN; MESSENGER RNA PRECURSOR; OLIGONUCLEOTIDE; PARVOVIRUS VECTOR; SPLICE SWITCHING OLIGONUCLEOTIDE; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ARTICLE; CONTROLLED STUDY; FOOD AND DRUG ADMINISTRATION; GENE MUTATION; MOUSE; NONHUMAN; PHOTORECEPTOR; PRIORITY JOURNAL; RETINA CELL; RNA SPLICING; UNITED STATES; WILD TYPE MOUSE;

EID: 84940834150     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2015.24     Document Type: Article

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