Generation of the induced pluripotent stem cell line (ZSPHARi001-A) from a patient with recessive dystrophic epidermolysis bullosa carrying compound heterozygous mutation in the COL7A1 gene.

The COL7A1 gene mutation causes type VII collagen dysfunction, which subsequently leads to recessive dystrophic epidermolysis bullosa (RDEB). Patients who suffer from RDEB experience severe blisters and chronic trauma, which can eventually result in serious infection and the development of fatal squamous cell carcinoma. In our study, peripheral blood mononuclear cells (PBMCs) from an RDEB patient with the COL7A1 compound heterozygous mutation were collected and then reprogrammed into induced pluripotent stem cells (iPSC). The RDEB iPSC line can provide a cellular resource for the study of pathogenesis and drug screening.

NIR-Responsive Copolymer Upconversion Nanocomposites for Triggered Drug Release in Vitro and in Vivo.

Light has several advantages as the stimulus for a triggered drug release. Currently, the applications of phototriggered drug-release devices (PDDs) are largely limited by two factors: the limited tissue penetration and detrimental effects caused by excitation light (ultraviolet or visible light). To address this disadvantage, this study developed nanocomposites based on upconversion nanoparticles (UC), which could convert near-infrared light to ultraviolet-visible light and trigger drug release. By loading UC and doxorubicin (DOX) into photoresponsive copolymer PEG-NMAB-PLA (PNP), near-infrared responsive copolymer upconversion nanocomposites (PNP-DOX-UC) were constructed. We proved that PNP-DOX-UC showed the fast release and strong cytotoxicity under near-infrared irradiation in vitro. The therapeutic efficacy study indicated that PNP-DOX-UC+hv had the enhanced antitumor efficiency. In the study, UC becoming an internal ultraviolet-visible light source for near-infrared excitation develops an applicable and efficient approach to meet the requirements for UV/vis excitation, which is a major disadvantage in photosensitive materials developed for pharmaceutical and biomedical applications.