Antisense oligonucleotide (AON)-based therapy for leber congenital amaurosis caused by a frequent mutation in CEP290

Molecular Therapy Nucleic Acids

Volumn 1, Issue 3, 2012, Pages e14-

  Collin, Rob Wj ^a,b   Den Hollander, Anneke I ^a,b   Der Velde Visser, Saskia D Van ^a   Bennicelli, Jeannette ^c   Bennett, Jean ^c   Cremers, Frans Pm ^a,b  

^a RADBOUD UNIVERSITY MEDICAL CENTER   (Netherlands)

^b RADBOUD UNIVERSITY MEDICAL CENTER   (Netherlands)

^c UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

Antisense oligonucleotides; AON; CEP290; Genetic therapy; LCA

Indexed keywords

EID: 84867129125     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2012.3     Document Type: Article

References (40)

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