Generation of mutation-corrected induced pluripotent stem cell lines derived from adrenoleukodystrophy patient by using homology directed repair.

X-linked adrenoleukodystrophy (ALD) caused by the ABCD1 mutation, is the most common inherited peroxisomal disease. Previously, we generated an ALD patient-derived SCHi001-A iPSC model. In this study, we have performed the first genome editing of ALD patient-derived SCHi001-A iPSCs using homology-directed repair (HDR). The mutation site, c.1534G > A [GenBank: NM_000033.4], was corrected by introducing ssODN and the CRISPR/Cas9 system. The cell line exhibited normal iPSC plulipotency marker expression following genome editing. Mutation-corrected iPSCs from SCHi001-A iPSC line can be used in research into the pathophysiology of and therapeutics for ALD.

Clinical feasibility of brain-computer interface based on steady-state visual evoked potential in patients with locked-in syndrome: Case studies.

Although the feasibility of brain-computer interface (BCI) systems based on steady-state visual evoked potential (SSVEP) has been extensively investigated, only a few studies have evaluated its clinical feasibility in patients with locked-in syndrome (LIS), who are the main targets of BCI technology. The main objective of this case report was to share our experiences of SSVEP-based BCI experiments involving five patients with LIS, thereby providing researchers with useful information that can potentially help them to design BCI experiments for patients with LIS. In our experiments, a four-class online SSVEP-based BCI system was implemented and applied to four of five patients repeatedly on multiple days to investigate its test-retest reliability. In the last experiments with two of the four patients, the practical usability of our BCI system was tested using a questionnaire survey. All five patients showed clear and distinct SSVEP responses at all four fundamental stimulation frequencies (6, 6.66, 7.5, 10 Hz), and responses at harmonic frequencies were also observed in three patients. Mean classification accuracy was 76.99% (chance level = 25%). The test-retest reliability experiments demonstrated stable performance of our BCI system over different days even when the initial experimental settings (e.g., electrode configuration, fixation time, visual angle) used in the first experiment were used without significant modifications. Our results suggest that SSVEP-based BCI paradigms might be successfully used to implement clinically feasible BCI systems for severely paralyzed patients.

New schiartane-type triterpene from Kadsura heteroclita and their cytotoxic activities.

One new schiartane-type triterpene, heteroclitalactone N (1), and four known compounds (2-5), seco-coccinic acid F, dihydroguaiaretic acid, schizanrin F, and kadsuralignan B were isolated from the stems of Kadsura heteroclita. Their structures were determined by the combination of spectroscopic and chemical methods, including 1D-, 2D-NMR, and CD spectra as well as by comparing with the NMR data reported in the literature. The cytotoxic activities of all isolated compounds were evaluated on three human cancer cell lines. Compound 3 exhibited moderate to weak cytotoxic activities on three human cancer cell lines, OVCAR, HT-29, and A-549, with IC50 values ranging from 16.2 to 36.4 microM.

New butenolide and pentenolide from Dysidea cinerea.

Two new butenolide and pentenolide derivatives, dysideolides A-B, were isolated from the marine sponge Dysidea cinerea. Their structures were determined by the combination of spectroscopic and chemical methods, including 1D- and 2D-NMR spectroscopy, and CD spectra, as well as by comparing with the NMR data reported in the literature.