Development of treatments for deafness and blindness in Usher Syndrome


we are developing approaches to treat usher syndrome by manipulating pre-mrna splicing Using Antisense OligoNucleotides

Movie of Usher and non-Usher mice. (Mutant(AA)) treated with an untargeted ASO (Mutant(AA)- ASO-C; lower left) have vestibular dysfunction as evidenced by the circling behavior. These mice are also profoundly deaf. Usher mice treated with the ASO targeted to correct expression of the 216A mutated Ush1c gene (Mutant(AA) ASO-29; lower right) exhibit normal behavior indicating correction of balance. Hearing is also rescued in these mice. Mice with only one copy of the mutation (Het (GA); top) can hear and have normal balance and are indistinguishable from the treated mutant mice.


Check out our Latest study to deliver Antisense Oligonucleotides in Utero by injection into the amniotic cavity.