The potential of small molecule brain-derived neurotrophic factor: mimetics to treat inherited retinal degeneration

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Title: The potential of small molecule brain-derived neurotrophic factor: mimetics to treat inherited retinal degeneration
Authors: Cerquone Perpetuini, Andrea
Mathoux, Justine
Kennedy, Breandán
Permanent link: http://hdl.handle.net/10197/9611
Date: Jan-2019
Online since: 2019-02-25T11:35:28Z
Abstract: Is there a need for small molecule neuroprotectants in inherited retinal degeneration (iRDs)? iRDs are a heterogeneous cluster of diseases which lead to blindness in 1 in every 2–3000 people. A plethora of causative genetic defects were revealed in the last 30 years. Current research focuses on development of pharmacological, biological or mechanical implant treatments. Gene therapy demonstrated success in patients with specific genetic mutations and Luxturna, recently approved by the FDA for RPE65 related disease, entered the market at a cost of $425,000 per eye.
Funding Details: Enterprise Ireland
European Commission Horizon 2020
Science Foundation Ireland
Type of material: Journal Article
Publisher: Wolters Klumer
Journal: Neural Regeneration Research
Volume: 14
Issue: 1
Start page: 85
End page: 86
Copyright (published version): 2018 the Authors
Keywords: Gene therapyNeurotrophic factorsRetinal degeneration
DOI: 10.4103/1673-5374.243711
Language: en
Status of Item: Peer reviewed
Appears in Collections:Biomolecular and Biomedical Science Research Collection

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