This week in therapeutics




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Hearing loss

Usher syndrome 1C (USH1C)

Mouse studies suggest using antisense oligonucleotides (ASOs) that correct splicing of mutant USH1C transcripts could help prevent Usher syndrome-associated hearing loss. In a neonatal mouse model of Usher syndrome, a single intraperitoneal injection of a splice-correcting ASO prevented development of auditory and vestibular defects, whereas injection of a control ASO did not. Next steps include optimizing the lead ASO and determining the best dosing regimen in collaboration with Isis Pharmaceuticals Inc.

SciBX 6(7); doi:10.1038/scibx.2013.171
Published online Feb. 21, 2013

Patents pending covering ASOs targeted to the mutant USH1C; licensing status undisclosed

Lentz, J.J. et al. Nat. Med.;
published online Feb. 4, 2013;
Contact: Michelle L. Hastings,
Rosalind Franklin University of Medicine and Science, Chicago, Ill.