학술논문
RNA interference-mediated suppression and replacement of human rhodopsin in vivo
Document Type
Academic Journal
Author
Source
American Journal of Human Genetics. July, 2007, Vol. 81 Issue 1, p127, 9 p.
Subject
Language
English
ISSN
0002-9297
Abstract
The investigation of therapeutics comprising of gene suppression in conjunction with gene replacement is presented. The results have shown that suppression and replacement can provide a therapeutic solution for dominantly inherited disorders such as rhodopsin gene (RHO)-linked retinitis pigmentosa (RP) and can be used to circumvent mutational heterogeneity.