Olfactory loss and dysfunction in ciliopathies: Molecular mechanisms and potential therapies.

Olfactory loss and dysfunction in ciliopathies: Molecular mechanisms and potential therapies.
Curr Med Chem. 2018 Jan 04;:
Authors: Uytingco CR, Green WW, Martens JR
Abstract
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 of ciliopathies in the olfactory system. Importantly, it will highlight both pharmacological and biological approaches, and their potential therapeutic value in the olfactory system and other ciliated tissues.
PMID: 29303074 [PubMed - as supplied by publisher]

Source: Current Medicinal Chemistry - January 4, 2018 Category: Chemistry Authors: Uytingco CR, Green WW, Martens JR Tags: Curr Med Chem Source Type: research