Merve Sen

TL;DR: This article used magnetic nanoparticles (MNPs) and reverse magnetofection (Reverse Magnetofection) to deliver siRNA/MNP complexes into retinal explant tissue, targeting valosin-containing protein (VCP) previously established as a potential therapeutic target for autosomal dominant retinitis pigmentosa (adRP).

TL;DR: This work provides the first evidence that inhibition of VCP activity rescues degenerating P23H rod cells and improves their functional properties in P 23H transgenic rat and P23h knock-in mouse retinae, both in vitro and in vivo.

TL;DR: In this paper, two nano-formulations for the VCP inhibitor ML240 were obtained by using amphiphilic polymers methoxy-poly (ethylene glycol)5kDa-cholane (mPEG5k Da-choline) and methoxypoly(ethylene- glycol), 5kDa -choline (mPG5kda-cholesterol), respectively, to increase the water-solubility of ML240 by two orders of magnitude.

TL;DR: In this article, the authors investigated and compared pharmacological modulation of ERAD at four different major steps, including protein recognition, targeting for ERAD, retrotranslocation, and proteasomal degradation, and showed that inhibition of the VCP/proteasome activity favors cell survival and suppresses P23H-mediated retinal degeneration in rat retinal explants.

TL;DR: In this paper, the authors 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.