2019
DOI: 10.1016/j.preteyeres.2018.08.003
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Gene therapy for visual loss: Opportunities and concerns

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Cited by 81 publications
(59 citation statements)
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“…The immune privilege of the eye makes it suitable targets for AAV‐mediated gene therapy. [ 8d,10b,25 ] Previous studies showed that Cas9 nuclease could generate double‐strand breaks (DSBs) to disrupt genes function in the retina. [ 26 ] Precise editing of retinal cells by HDR was reported in newborn mice, but editing efficiency was low (<3%).…”
Section: Resultsmentioning
confidence: 99%
“…The immune privilege of the eye makes it suitable targets for AAV‐mediated gene therapy. [ 8d,10b,25 ] Previous studies showed that Cas9 nuclease could generate double‐strand breaks (DSBs) to disrupt genes function in the retina. [ 26 ] Precise editing of retinal cells by HDR was reported in newborn mice, but editing efficiency was low (<3%).…”
Section: Resultsmentioning
confidence: 99%
“…However, adverse effects associated with prolonged expression of vascular targeting proteins by gene delivery may have unwanted side effects, including retinal vascular toxicity. There are also other major obstacles to the acceleration of gene therapy from bench to bedside, such as cost of the treatment [ 19 ]. Therefore, in the present study, we rationally designed a small fragment of CAD, CAD27, which covers the core anti-angiogenic domain of CAD and can be produced by chemical synthesis at a lower cost than its parent molecule, recombinant CAD protein or CAD gene therapy.…”
Section: Discussionmentioning
confidence: 99%
“…However, adverse effects associated with prolonged expression of vascular targeting proteins by gene delivery may have unwanted side effects, including retinal vascular toxicity. There are also other major obstacles to the acceleration of gene therapy from bench to bedside, such as cost of the treatment [19]. Therefore, in the present study, we rationally designed a small fragment of CAD, CAD27, which covers the core antiangiogenic domain of CAD and can be produced by chemically synthesis at a lower cost than its parent molecule, recombinant CAD protein or CAD gene therapy.…”
Section: Discussionmentioning
confidence: 99%