In vivoversusex vivoCRISPR therapies for retinal dystrophy.
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 cellsex vivofor autologous transplant, whereas the second is to modify cellsin vivoby 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'sAudacious Goals Initiative. The clinical translatability of each path is discussed with regard to current investigations and whether cell replacement can be circumvented byin vivoediting.
Board of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Benjamin
B
Bakondi
CRISPR; gene editing; retinal degeneration; retinal dystrophy; retinitis pigmentosa; translational medicine