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Smelling the roses and seeing the light: gene therapy for ciliopathies

Paper ID Volume ID Publish Year Pages File Format Full-Text
36991 45294 2013 9 PDF Available
Title
Smelling the roses and seeing the light: gene therapy for ciliopathies
Abstract

•Gene therapies have been successfully used in several mammalian ciliopathy models.•Rescued defects include cilia mobility, vision, and olfaction.•Overcoming challenges presented by pleiotropy remains a goal for effective treatments.•Therapies providing palliative care offer substantial benefits to patients.

Alterations in cilia formation or function underlie a growing class of pleiotropic disorders termed ciliopathies. The genetic basis of ciliopathies is remarkably complex, with an incomplete but expanding list of more than 89 loci implicated in various disorders. Current treatment of ciliopathies is limited to symptomatic therapy. However, our growing understanding of ciliopathy genetics, coupled with recent advances in gene delivery and endogenous gene and transcript repair demonstrated thus far in tissues of the eye, nose, and airway, offers hope for curative measures in the near future. This review highlights these advances, as well as the challenges that remain with the development of personalized medicine for treating a very complex spectrum of disease, penetrant in a variety of organ systems.

Keywords
cilia; viral vectors; genetic disorder; gene therapy; ciliopathies; treatment
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Publisher
Database: Elsevier - ScienceDirect
Journal: - Volume 31, Issue 6, June 2013, Pages 355–363
Authors
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Subjects
Physical Sciences and Engineering Chemical Engineering Bioengineering
Get Full-Text Now
Don't Miss Today's Special Offer
Price was $35.95
You save - $31
Price after discount Only $4.95
100% Money Back Guarantee
Full-text PDF Download
Online Support
Any Questions? feel free to contact us