title: Cell and gene therapy.
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.
authors.affiliation: Department of Pathology, University of Michigan Medical School, Ann Arbor, Mich., USA.
authors.forename: Rajesh C
authors.initials: RC
authors.lastname: Rao
authors.forename: David N
authors.initials: DN
authors.lastname: Zacks
mesh_terms: D000818:Animals; D064987:Cell- and Tissue-Based Therapy / Q000379:methods*; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D012162:Retinal Degeneration / Q000628:therapy*
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