Michelle L. Hastings, Ph.D.
The Research
Rosalind Franklin University of Medicine and Science
Therapeutic correction of Ush1c splicing in a mouse model of usher syndrome
Usher syndrome is the leading genetic cause of combined hearing and vision loss. The long-term objective of this project is to develop therapeutics for the disease. Antisense oligonucleotides (ASOs) will be used in a mouse model of Usher syndrome to correct a specific genetic defect that causes the disease. This work will demonstrate the efficacy of ASOs as a therapeutic for Usher syndrome and will also provide insights about curing the disease.