Article
Genomic DNA nanoparticles rescue rhodopsin-associated retinitis pigmentosa phenotype.
FASEB journal : official publication of the Federation of American Societies for Experimental Biology - 1 Jun 2015
Han Zongchao, Banworth Marcellus J, Makkia Rasha, Conley Shannon M, Al-Ubaidi Muayyad R, Cooper Mark J, Naash Muna I
Abstract excerpt
Mutations in the rhodopsin gene cause retinal degeneration and clinical phenotypes including retinitis pigmentosa (RP) and congenital stationary night blindness. Effective gene therapies have been difficult to develop, however, because generating precise levels of rhodopsin expression is critical; overexpression causes toxicity, and underexpression would result in incomplete rescue. Current gene delivery...
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