RNAi in Ocular Diseases

Turchinovich, Andrey; Zoidl, Georg; Dermietzel, Rolf

doi:10.1002/9781118610749.ch18

Cited by 2 publications

(3 citation statements)

References 95 publications

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“…RNAi therapies treating anterior ophthalmic disease avoids the challenges of targeted delivery and bioavailability present for other indications because of the easy accessibility and sequestered structure of the anterior ocular segment 94 . Notably, a recently developed reducible branched polyethylenimine (rBPEI)-based nanoparticle (NP) system was able to deliver siRNA throughout the cornea to treat corneal neovascularization in rats 73,95 .…”

Section: Epigenetic Therapiesmentioning

confidence: 99%

Epigenetic regulation of anterior segment diseases and potential therapeutics

et al. 2020

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In recent years, technological advances in sequencing have accelerated our understanding of epigenetics in ocular development and ophthalmic diseases. We now know that epigenetic modifications are necessary for normal ocular development and biological processes such as corneal wound healing and ocular surface repair, while aberrant epigenetic regulation underlies the pathogenesis of a wide range of ocular diseases, including cataracts and various diseases of the ocular surface. As the epigenetics of the eye is a constantly changing field of medicine, this comprehensive review focuses on innovations and scientific discoveries related to epigenetic control of anterior segment diseases that were published in the English literature in the past five years. These recent studies attempt to elucidate therapeutic targets for the anterior segment pathological processes. Already, recent studies have shown therapeutic potential in targeting epigenetic mechanisms of ocular disease, and new epigenetic therapies are on the verge of being introduced to clinical practice. New drug targets can potentially emerge as we make further discoveries within this field.

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Section: Epigenetic Therapiesmentioning

confidence: 99%

Epigenetic regulation of anterior segment diseases and potential therapeutics

et al. 2020

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“…These numbers are expected to double by 2050 . In the spectrum of the possible strategies to address these pathologies, the silencing of specific genes via small interfering RNA (siRNA) delivery holds huge potential. , This is especially true for diseases that have been linked to anomalous activities of defined proteins. Chronic diseases such as some forms of retinitis pigmentosa, age-related macular degeneration (AMD), diabetic retinopathy, and glaucoma might benefit from this approach.…”

Section: Introductionmentioning

confidence: 99%

“…Furthermore, siRNAs per se do not cross the cellular membrane of intact cells. To tackle this problem, nonviral vectors (i.e., cationic lipids, polymers, peptides, or nanoparticles) as well as physical methods of transfection (electroporation, ultrasound) or combinations of the two , have been developed and indeed improved the siRNA ocular pharmacodynamic in vivo . Yet, their application is not free from the need of invasive injections such as intravitreal injections. , Furthermore, the genetic modification of retinal cells occurs in the sole layer that directly contacts the vitreous, as reported from the large majority of studies using both nonviral − and viral , vectors on clinically relevant animal models.…”

Section: Introductionmentioning

confidence: 99%

New Method for Efficient siRNA Delivery in Retina Explants: Reverse Magnetofection

et al. 2021

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The prevalence of retinal disorders associated with visual impairment and blindness is increasing worldwide, while most of them remain without effective treatment. Pharmacological and molecular therapy development is hampered by the lack of effective drug delivery into the posterior segment of the eye. Among molecular approaches, RNA-interference (RNAi) features strong advantages, yet delivering it to the inner layer of the retina appears extremely challenging. To address this, we developed an original magnetic nanoparticles (MNPs)-based transfection method that allows the efficient delivery of siRNA in all retinal layers of rat adult retinas through magnetic targeting. To establish delivery of RNAi throughout the retina, we have chosen organotypic retinal explants as an ex vivo model and for future high content screening of molecular drugs. Conversely to classic Magnetofection, and similar to conditions in the posterior chamber of the eye, our methods allows attraction of siRNA complexed to MNPs from the culture media into the explant. Our method termed “Reverse Magnetofection” provides a novel and nontoxic strategy for RNAi-based molecular as well as gene therapy in the retina that can be transferred to a wide variety of organ explants.

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RNAi in Ocular Diseases

Cited by 2 publications

References 95 publications

Epigenetic regulation of anterior segment diseases and potential therapeutics

Epigenetic regulation of anterior segment diseases and potential therapeutics

New Method for Efficient siRNA Delivery in Retina Explants: Reverse Magnetofection

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