TY - JOUR T1 - Genetic manipulation for inherited neurodegenerative diseases: myth or reality? JF - British Journal of Ophthalmology JO - Br J Ophthalmol SP - 1322 LP - 1331 DO - 10.1136/bjophthalmol-2015-308329 VL - 100 IS - 10 AU - Patrick Yu-Wai-Man Y1 - 2016/10/01 UR - http://bjo.bmj.com/content/100/10/1322.abstract N2 - Rare genetic diseases affect about 7% of the general population and over 7000 distinct clinical syndromes have been described with the majority being due to single gene defects. This review will provide a critical overview of genetic strategies that are being pioneered to halt or reverse disease progression in inherited neurodegenerative diseases. This field of research covers a vast area and only the most promising treatment paradigms will be discussed with a particular focus on inherited eye diseases, which have paved the way for innovative gene therapy paradigms, and mitochondrial diseases, which are currently generating a lot of debate centred on the bioethics of germline manipulation. ER -