DB-OTO is an experimental

gene therapy Gene therapy is Health technology, medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells. The first attempt at modifying human DNA ...

for

otoferlin Otoferlin is a protein that in humans is encoded by the ''OTOF'' gene. It is involved in vesicle membrane fusion, and mutations in the OTOF gene are associated with a genetic form of deafness. Function There are two forms of otoferlin protein ...

-related hearing loss developed by

Regeneron Pharmaceuticals Regeneron Pharmaceuticals, Inc. is an American biotechnology company headquartered in Westchester County, New York. The company was founded in 1988. Originally focused on neurotrophic factors and their regenerative capabilities, giving rise to ...

. It is delivered via

adeno-associated virus Adeno-associated viruses (AAV) are small viruses that infect humans and some other primate species. They belong to the genus '' Dependoparvovirus'', which in turn belongs to the family ''Parvoviridae''. They are small (approximately 26 nm in ...

References

{{reflist Experimental gene therapies