Olfactory Loss and Dysfunction in Ciliopathies: Molecular Mechanisms and Potential Therapies.

ABSTRACT:

Background

Ciliopathies are a class of inherited pleiotropic genetic disorders in which alterations in cilia assembly, maintenance, and/or function exhibit penetrance in the multiple organ systems. Olfactory dysfunction is one such clinical manifestation that has been shown in both patients and model organisms. Existing therapies for ciliopathies are limited to the treatment or management of symptoms. The last decade has seen an increase in potential curative therapeutic options including small molecules and biologics. Recent work in multiciliated olfactory sensory neurons has demonstrated the capacity of targeted gene therapy to restore ciliation in terminally differentiated cells and rescue olfactory function. This review will discuss the current understanding of the penetrance o

SUBMITTER: Uytingco CR 

PROVIDER: S-EPMC6034980 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature