Key result
Dual-AAV delivery of the 6 kb full-length otoferlin cDNA into inner hair cells successfully restored hearing in otoferlin knock-out mice.
Why the study?
AAV application is limited by a low cargo capacity restricting use to sequences <4 kb, prompting approaches like dual-AAV vectors to deliver larger genes.
Population
Otoferlin knock-out mice
Design
Review
Authors
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Does not support clinical use; extends dual-AAV feasibility for large genes in preclinical inner-ear models.
Dual-AAV vectors can successfully deliver large gene sequences (>4 kb) to the inner ear, paving the way for gene therapies for deafness caused by large gene mutations.
Ellen Reisinger (2019) conducted a review in Deafness. Dual-AAV delivery of large gene sequences was evaluated on Restoration of hearing. Dual-AAV delivery of the 6 kb full-length otoferlin cDNA into inner hair cells successfully restored hearing in otoferlin knock-out mice.
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