Titlebook: Recent Advances In Retinal Degeneration; Robert E. Anderson,Matthew M. LaVail,Joe G. Hollyf Book 2008 The Editor(s) (if applicable) and Th

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In vitro Analysis of Ribozyme-mediated Knockdown of an ADRP Associated Rhodopsin Mutationsub divided into 3 classes, autosomal dominant, autosomal recessive, and X- linked RP, where the mutant gene exists on the sex chromosome (X) (Hartong et al., 2006; Wang et al., 2005). Mutations in rhodopsin are the most common cause of the autosomaldominant form of RP (ADRP). More than 100 mutation

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Development of Viral Vectors with Optimal Transgene Expression for Ocular Gene Therapiesrapies using viral vectors are one of the promising technologies. For an ideal therapeutic effect, a variety of vector designs have been tested. Especially, selection of a promoter to drive a transgene is important to obtain adequate level of expression. In this brief chapter, reviewed are the promo

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Adeno-Associated Viral Vectors and the RetinaThe retina is a prime location for gene therapy because of its accessibility, immune privileged status (Caspi, 2006), and susceptible cell types. Several strategies have been attempted to rescue retinal disease, including gene replacement (Acland et al., 2001), gene knockdown with both ribozymes (Go

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