Antisense oligonucleotides targeting mutant Ataxin-7 restore visual function in a mouse model of spinocerebellar ataxia type 7

• DOI: 10.1126/scitranslmed.aap8677
• Publication Date: 2018-10-31T18:09:29Z
• Authors: Chenchen Niu; Thazah P. Prakash; Aneeza Kim; John L. Quach; Laryssa A. Huryn; Yuechen Yang; Edith Lopez; Ali Jazayeri; Gene Hung; Bryce L. Sopher; Brian P. Brooks; Eric E. Swayze; C. Frank Bennett; Albert R. La Spada
• Keywords: Vision; Oligonucleotides; Retina; Epigenesis, Genetic; Mice; Protein Aggregates; 03 medical and health sciences; Genetic; Ocular; Animals; Humans; Spinocerebellar Ataxias; Photoreceptor Cells; Antisense; Vision, Ocular; Ataxin-7; 0303 health sciences; Animal; Vertebrate; Retinal Degeneration; Oligonucleotides, Antisense; Chromatin Assembly and Disassembly; Disease Models, Animal; Phenotype; Gene Expression Regulation; Disease Models; Intravitreal Injections; Disease Progression; Mutant Proteins; Peptides; Epigenesis; Photoreceptor Cells, Vertebrate

Intravitreal injection of antisense oligonucleotides targeting mutated Ataxin-7 improves visual function in a mouse model of spinocerebellar ataxia type 7.