Nanoparticle‐motivated gene delivery for ophthalmic application

Abstract

Ophthalmic gene therapy is an intellectual and intentional manipulation of desired gene expression into the specific cells of an eye for the treatment of ophthalmic (ocular) genetic dystrophies and pathological conditions. Exogenous nucleic acids such as DNA, small interfering RNA (siRNA), micro RNA (miRNA), etc., are used for the purpose of managing expression of the desired therapeutic proteins in ocular tissues. The delivery of unprotected nucleic acids into the cells is limited due to exogenous and endogenous degradation modalities. Nanotechnology, a promising and sophisticated cutting edge tool, works as a protective shelter for these therapeutic nucleic acids. They are able to be safely delivered to the required cells in order to modulate anticipated protein expression. To this end, nanotechnology is seen as a potential and promising strategy in the field of ocular gene delivery. This review focused on current nanotechnology modalities and other promising non-viral strategies being used to deliver therapeutic genes in order to treat various devastating ocular diseases.

Neuroprotective Effect of siRNA Entrapped in Hyaluronic Acid-Coated Lipoplexes by Intravitreal Administration

“…Viral vectors and non-viral vectors are two main approaches used for siRNA or gene transfer in ocular applications [17,18]. The most used viral vectors for retinal gene transfer are those derived from adenoviruses, lentiviruses and adeno-associated viruses, which can infect and transduce non-dividing cells, such as photoreceptors and the retinal pigment epithelium (RPE) [12,[19][20][21].…”

Injectable hydrogels for ophthalmic applications

“…adeno-associated virus (AAV) and lentivirus, are highly effective transfection tools for gene therapy in ophthalmic research. However, this type of delivery vehicle has underlying safety concerns of immunogenicity, broad tissue tropism, carcinogenesis, and genomic insertional mutagenesis in clinical trials, thus in the end it is hoped that physicians and patients will accept only non-viral vectors as the standard of treatment [14].…”

Low-Fouling Characteristics of Ultrathin Zwitterionic Cysteine SAMs

“…Recent methodologies extend the lifetime of implantable devices by minimizing the adsorption of nonspecific plasma proteins at the initial stage of implantation. ,, Often, the surfaces are functionalized with self-assembled zwitterionic molecules, which form a tightly solvated layer of water molecules, , to impart antifouling capabilities. − Compared to conventional hydrophilic polymers, such as polyethylene glycol, many recently developed zwitterionic surfaces exhibit superior performance in resisting protein fouling. ,, Early work by Jiang and co-workers has demonstrated the feasibility of using carboxybetaine and sulfobetaine for zwitterionic antifouling materials. − Chen et al have reported ultralow protein adsorption (<0.3 ng/cm 2 ) on surfaces functionalized with a layer of alternating Glu/Lys or Asp/Lys . Moreover, the antifouling properties and long blood circulation characteristics of zwitterions have been integrated recently with quantum dots, hydrogels, silica nanoparticles, , and liposomes to improve functionality across a range of biological applications.…”

“…29−31 Chen et al have reported ultralow protein adsorption (<0.3 ng/cm 2 ) on surfaces functionalized with a layer of alternating Glu/Lys or Asp/Lys. 28 Moreover, the antifouling properties and long blood circulation characteristics of zwitterions have been integrated recently with quantum dots, 32 hydrogels, 33 silica nanoparticles, 34,35 and liposomes 36 to improve functionality across a range of biological applications.…”