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Review
. 2014:53:167-77.
doi: 10.1159/000357376. Epub 2014 Apr 10.

Cell and gene therapy

Rajesh C Rao  1 , David N Zacks
Affiliations
Review

Cell and gene therapy

Rajesh C Rao et al. Dev Ophthalmol. 2014.

Abstract

Replacement or repair of a dysfunctional gene combined with promoting cell survival is a two-pronged approach that addresses an unmet need in the therapy of retinal degenerative diseases. In this chapter, we discuss various strategies toward achieving both goals: transplantation of wild-type cells to replace degenerating cells and to rescue gene function, sequential gene and cell therapy, and in vivo reprogramming of rods to cones. These approaches highlight cutting-edge advances in cell and gene therapy, and cellular lineage conversion in order to devise new therapies for various retinal degenerative diseases.

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