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2015 ; 10
(3
): 281-299
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Retinal Gene Therapy: Current Progress and Future Prospects
#MMPMID26609316
Ku CA
; Pennesi ME
Expert Rev Ophthalmol
2015[Jun]; 10
(3
): 281-299
PMID26609316
show ga
Clinical trials treating inherited retinal dystrophy caused by RPE65 mutations
had put retinal gene therapy at the forefront of gene therapy. Both successes and
limitations in these clinical trials have fueled developments in gene vectors,
which continue to further advance the field. These novel gene vectors aim to more
safely and efficiently transduce retinal cells, expand the gene packaging
capacity of AAV, and utilize new strategies to correct the varying mechanisms of
dysfunction found with inherited retinal dystrophies. With recent clinical trials
and numerous pre-clinical studies utilizing these novel vectors, the future of
ocular gene therapy continues to hold vast potential.