Purpose: Inherited retinal dystrophies are a significant cause of vision loss and are
characterized by the loss of photoreceptors and the retinal pigment epithelium (RPE) …

US9938521B2 - CRISPR/CAS-related methods and compositions for treating leber's
congenital amaurosis 10 (LCA10) - Google Patents US9938521B2 - CRISPR/CAS-related …

The advent of gene editing has introduced the ability to make changes to the genome of
cells, thus allowing for correction of genetic mutations in patients with monogenic diseases …

The human photoreceptor function is dependent on a highly specialised cilium. Perturbation
of cilial function can often lead to death of the photoreceptor and loss of vision. Retinal …

Purpose: Recent concerns regarding the clinical utilization of clustered regularly interspaced
short palindromic repeats (CRISPR) involve uncertainties about the potential detrimental …

Abstract Purpose of Review Clustered regularly interspaced short palindromic repeats
(CRISPR) system is a genome engineering system with great potential for clinical …

Introduction: Genetic alterations resulting in a dysfunctional retinal pigment epithelium
and/or degenerating photoreceptors cause impaired vision. These juxtaposed cells in the …

Two therapeutic paths have been proposed to treat inherited retinal dystrophy using
clustered regularly interspaced short palindromic repeats (CRISPR). One strategy is to …

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compound O= C [C@ H]([U]) O [C@@ H](C= O) CO KMASKRIRDRONCR-JEDNCBNOSA-N …

Inherited retinal diseases are considered as a leading cause of vision loss in a young
population. Neither a permanent cure nor long-term treatment has yet been discovered …