In vivo versus ex vivo CRISPR therapies for retinal dystrophy
B Bakondi - Expert review of ophthalmology, 2016 - Taylor & Francis
Expert review of ophthalmology, 2016•Taylor & Francis
Two therapeutic paths have been proposed to treat inherited retinal dystrophy using
clustered regularly interspaced short palindromic repeats (CRISPR). One strategy is to
genetically correct patient cells ex vivo for autologous transplant, whereas the second is to
modify cells in vivo by delivering CRISPR effectors to the retina. The feasibility of both
editing strategies has been demonstrated within three years of CRISPR's adaptation to
mammalian systems. However, the functional integration of transplanted cells into host …
clustered regularly interspaced short palindromic repeats (CRISPR). One strategy is to
genetically correct patient cells ex vivo for autologous transplant, whereas the second is to
modify cells in vivo by delivering CRISPR effectors to the retina. The feasibility of both
editing strategies has been demonstrated within three years of CRISPR's adaptation to
mammalian systems. However, the functional integration of transplanted cells into host …
Summary
Two therapeutic paths have been proposed to treat inherited retinal dystrophy using clustered regularly interspaced short palindromic repeats (CRISPR). One strategy is to genetically correct patient cells ex vivo for autologous transplant, whereas the second is to modify cells in vivo by delivering CRISPR effectors to the retina. The feasibility of both editing strategies has been demonstrated within three years of CRISPR’s adaptation to mammalian systems. However, the functional integration of transplanted cells into host retinae has been a long-standing challenge that currently represents the 2025 moonshot of the National Eye Institute’s Audacious Goals Initiative. The clinical translatability of each path is discussed with regard to current investigations and whether cell replacement can be circumvented by in vivo editing.
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